This is a multicenter prospective and retrospective observational clinical study in patients with primary or post polycythemia vera or post essential thrombocythemia myelofibrosis to test the efficacy of fedratinib in the rea world. Participants will be managed according to the clinical practice of the participating Center. All Centers will be Italian Hematology Units belonging to the GIMEMA Organization in Italy.

Start Date29 Jan 2024

Sponsor / Collaborator

Gruppo Italiano Malattie EMatologiche dell'Adulto

NCT06073847

/ RecruitingNot Applicable

Inrebic® (Fedratinib) Post-Marketing Surveillance in Korean Patients With Myelofibrosis

The purpose of this study is to assess the real-world safety of fedratinib for the treatment of adult participants with primary myelofibrosis (PMF), post polycythemia vera myelofibrosis (post-PV MF), or post essential thrombocythemia myelofibrosis (post-ET MF) who were previously treated with ruxolitinib. Participants will represent the overall patient population with PMF, post-PV MF or post-ET MF who lost adequate response to and/or are intolerant to ruxolitinib. Inadequate response definitions will follow Ministry of Food and Drug Safety-approved label and reimbursement criteria of the Health Insurance Review & Assessment Service.

Start Date13 Jun 2023

Sponsor / Collaborator

Bristol Myers Squibb Co.

CTIS2024-519227-22-00

/ Active, not recruitingPhase 1/2IIT

Fedratinib in Combination with CC-486, a Hypomethylating Agent, in Patients with Accelerated Phase Myelofibrosis - FAMy

Start Date03 Nov 2022

Sponsor / Collaborator

Martin-Luther-Universität Halle-Wittenberg

100 Clinical Results associated with Fedratinib Hydrochloride

100 Translational Medicine associated with Fedratinib Hydrochloride

100 Patents (Medical) associated with Fedratinib Hydrochloride

467

Literatures (Medical) associated with Fedratinib Hydrochloride

20 Jan 2026·ANALYTICAL CHEMISTRY

TIPP: A Novel Iodoacetamide Reaction-Induced Protein Precipitation Approach for Proteome-Wide Ligand-Target Identification

Article

Author: Zhang, Xiaolei ; Ma, Yanni ; Guan, Bangtong ; Ye, Mingliang ; Zhou, Jiahua ; Wang, Keyun ; Yan, Jiayang ; Wang, Jiayi

Ligand-bound proteins have higher stability and thereby greater resistance to denaturing-induced precipitation than free proteins, which forms the basis of several modification-free ligand target protein identification methods. However, due to the vast sequence diversity of cellular proteins, different proteins respond differently to distinct denaturation mechanisms, and a single denaturation mechanism can only cover certain target proteins. Therefore, developing efficient target protein identification methods based on novel denaturation mechanisms is of great significance for improving the coverage of target identification. Herein, we propose a novel target protein identification method relying on a new precipitation mechanism, namely, Target Identification by Iodoacetamide reaction-induced Protein Precipitation (TIPP). The TIPP method uses iodoacetamide (IAA), a thiol-blocking reagent commonly used in shotgun proteomics, to irreversibly alkylate the thiol groups of free cysteine residues, thereby inducing protein denaturation and precipitation. Under IAA treatment, proteins in different conformational states have different exposures of free thiol groups, resulting in different precipitation efficiencies, which is the key theoretical basis for the design of the TIPP method for target protein identifications. The feasibility of the developed TIPP method was evaluated by identifying target proteins of various ligand compounds, including MTX, TG101348, and staurosporine. In addition, this method was also used to monitor the target proteins of metal ions with the metal ion chelator TPEN. The developed method has similar efficiency and good complementarity with the traditional method, and can be combined with the traditional method to increase the coverage of target identification. Therefore, TIPP is expected to become a powerful strategy to achieve comprehensive ligand-target identification by complementing other drug target identification methods.

01 Jan 2026·BIOMEDICAL CHROMATOGRAPHY

Validated LC–MS/MS Method for the Quantitative Determination and Pharmacokinetic Profiling of Fedratinib (TG101348), an Oral JAK2 Inhibitor, in CD1 Mice Plasma

Article

Author: Boggavarapu, Rajesh Kumar ; Kandikere, Vishwottam ; Chimakurthy, Jithendra

ABSTRACT:

Fedratinib, a selective Janus kinase 2 (JAK2) inhibitor approved by the FDA for the treatment of myeloproliferative neoplasms, particularly myelofibrosis (MF), has clinical relevance. Despite this, no liquid chromatography–tandem mass spectrometry (LC–MS/MS) method has been reported for its quantification in biological matrices. This study aimed to develop and validate an LC–MS/MS method for quantifying fedratinib in mice plasma and assessing its pharmacokinetics. The method employed a Sciex 4500 triple quadrupole mass spectrometer with a Shimadzu LC system in positive ionization mode. Chromatographic separation was achieved using a Phenomenex Kinetex C18 column (2.1 × 50 mm, 5.0 μm) with a gradient mobile phase of 5 mM ammonium formate in 0.1% formic acid and acetonitrile at a flow rate of 0.8 mL/min. Telmisartan was used as the internal standard (IS). The method was validated in accordance with USFDA M10 bioanalytical guidelines. Fedratinib and IS were detected at m/z 525.5 → 468.9 and m/z 515.2 → 276.0, respectively. The assay demonstrated excellent sensitivity (LLOQ: 0.5 ng/mL), linearity (0.5 to 1000.0 ng/mL, r2 = 0.99), and accuracy (95.80–104.52%). Stability studies confirmed the analyte's integrity across multiple freeze–thaw cycles. The developed LC–MS/MS method is selective, sensitive, fully validated, and was successfully applied to pharmacokinetic studies.

31 Dec 2025·RENAL FAILURE

Fedratinib in chronic kidney disease: antifibrotic potential and renal safety signals from integrated network toxicology and pharmacovigilance

Article

Author: Han, Jinfen ; Bai, Zhixun ; Wang, Qin ; Zheng, Rubin ; Wang, Haiou ; Lyu, Jiayi ; Deng, Miao ; Chen, Jiaxi

Chronic kidney disease (CKD), a major global health challenge driven by renal fibrosis, lacks effective antifibrotic therapies. Fedratinib, a JAK2 inhibitor for myelofibrosis, shows kinase-modulating potential but uncharacterized utility and safety in CKD. Computational toxicity prediction utilized ProTox3.0 and ADMETlab2.0. Fedratinib-CKD targets were identified via combinatorial screening across databases (ChEMBL, STITCH, SwissTargetPrediction, GeneCards, OMIM, TTD). Core hubs were determined through protein-protein interaction network (STRING) and topological analysis (Cytoscape). Hierarchical docking validated binding (CB-Dock2). Post-marketing safety data (2019-2025) from FDA Adverse Event Reporting System (FAERS) was analyzed using disproportionality methods, including reporting odds ratio (ROR), proportional reporting ratio (PRR), Bayesian confidence propagation neural network (BCPNN) and multi-item gamma Poisson shrinker (MGPS). Computational profiling predicted severe respiratory, hepatic and cardiac toxicity but negligible nephrotoxicity. Network analysis identified 281 shared targets, with SRC, AKT1, EGFR and JAK2 as core hubs. Pathway enrichment revealed profound suppression of PI3K-Akt and MAPK signaling. Docking confirmed high-affinity binding. FAERS analysis of 1,268 reports identified significant renal and urinary disorders (ROR = 1.71) and extreme vitamin B1 deficiency (ROR = 1262.53), contradicting computational predictions. Fedratinib exhibits therapeutic duality: inhibition of SRC and JAK2 attenuates fibrosis by inhibiting JAK-STAT/TGFβ/Smad3 pathways, while off-target binding to AKT1/EGFR drives renal injury and vitamin B1 depletion-related toxicity with black-box encephalopathy warning. Evidence suggests for CKD patients with estimated glomerular filtration rate (eGFR) <45 mL/min/1.73 m² or age ≥70 years, a 50% dose reduction (200 mg/day) with renal function and thiamin monitoring (<30 nmol/L) may optimize risk-benefit profile.

News (Medical) associated with Fedratinib Hydrochloride

17 Feb 2026

·www.biospace.com

RECORDATI REPORTS STRONG PRELIMINARY FULL YEAR 2025 RESULTS: REVENUE +11.8%, EBITDA(1) +14.5%, ADJUSTED NET INCOME(2) +14.5%

Consolidated net revenue of € 2,618.4 million for full year 2025, +11.8% or +8.3% on a like-for-like basis (3) at constant exchange rates (CER) EBITDA (1) of € 991.1 million, +14.5%, margin on net revenue of 37.8% Adjusted net income (2) of € 651.1 million, +14.5%, margin on net revenue of 24.9% Net income of € 443.6 million, +6.5% Free cash flow (4) of € 558.8 million, +€ 23.7 million reflecting strong EBITDA partially offset by U.S. inventory build-up, higher interests and income tax paid Net debt (5) at € 2,037.3 million, just below 2.1x EBITDA FY 2026 financial targets: Net revenue € 2,730-€2,800 million with FX headwind of ~-3.5% ; EBITDA (1) € 995-€1,030 million, margin +/- 36.5% ; Adjusted net income (2) € 655-€ 685 million, margin +/- 24.0% In January 2026, the Group received a B‑ rating with ‘Prime’ status from ISS ESG, recognizing its leading sustainability performance within the industry Milan, February 17, 2026 – The Board of Directors of Recordati S.p.A. has reviewed and approved the preliminary consolidated financial statements for 2025. The Group’s final consolidated annual financial statements for 2025 will be submitted to the Board of Directors for approval on March 19, 2026. Rob Koremans, Chief Executive Officer of Recordati, commented: “2025 was another year of solid progress across the business, reflecting the strength of our execution. We delivered once again on our financial targets despite a challenging macroenvironment, including increased FX headwinds. During the year, we further strengthened our portfolio through strategic partnerships in both Rare Diseases and Specialty and Primary Care. There is excellent momentum in Rare Diseases, which continues to be a key driver of growth and value creation for the Group. We are excited by Isturisa’s opportunity to address the broader Cushing’s syndrome market, with uptake accelerating in the U.S. With such a strong foundation in place, we expect 2026 to be another year of disciplined execution as we continue to deliver on our strategic objectives, maintain sector-leading margins and create sustainable value for all our stakeholders.” Financial highlights Consolidated net revenue for full year 2025 was € 2,618.4 million, up 11.8% versus full year 2024 or 8.3% on a like-for-like (3) basis at CER, driven by solid contribution from both Specialty & Primary Care and Rare Diseases. The adverse FX impact for full year 2025 was € 64.2 million (-2.7%). Specialty & Primary Care revenue was € 1,477.9 million for full year 2025, up 2.0% or 3.8% on a like-for-like basis (3) at CER (+1.6% excluding Türkiye). This reflects continued positive performance of all core therapeutic areas (promoted product evolution index of 105), despite a slight slowdown in relevant market growth. In particular, the Urology and Cardiovascular franchises grew 2.5% and 2.8%, respectively, while the Gastrointestinal franchise grew 9.9%, driven by the strong in-market performance of several products in the portfolio, both prescription and OTC. Rare Diseases revenue was € 1,081.4 million for full year 2025, up 29.7% as compared to full year 2024, or 16.6% on a like-for-like (3) basis at CER, driven by strong volume growth across all three franchises. The Endocrinology franchise achieved net revenue of € 394.1 million, an increase of 22.5%, reflecting an acceleration of new patient uptake of Isturisa ® in the U.S. in the second half of 2025 with approximately 1,400 net active patients at the end of the year, as well as double-digit growth of Signifor ® . The Hema-Oncology franchise achieved net revenue of € 414.9 million, growing by 63.8%, reflecting the contribution of Enjaymo ® of € 146.3 million (+26.7% vs full year 2024 proforma (6) ), and driven by strong growth of Sylvant ® and Qarziba ® . The Metabolic franchise achieved net revenue of € 272.5 million, sustaining mid-single digit growth of 5.2%, driven by Carbaglu ® and Panhematin ® . Adjusted operating income (7) was € 774.9 million for full year 2025, up 13.2% over full year 2024, and 29.6% of net revenue versus 29.2% in the previous year. Operating income was € 670.8 million for full year 2025, up 5.0% over full year 2024, absorbing gross margin-related non-cash charges of € 66.8 million as compared to € 37.5 million for full year 2024, arising from the unwind of the fair value step up of acquired Rare Diseases inventory including € 62.5 million for Enjaymo ® . Non-recurring costs were € 37.3 million for full year 2025, versus € 8.0 million for full year 2024. These costs reflect primarily the continued optimization of the Specialty and Primary Care commercial organization, mainly in Italy and Spain. The non‑recurring costs also include a one‑off provision of €12.8 million (8) related to the settlement of a litigation case with AIFA concerning prior years payback for Urorec ® . Additionally, non‑recurring items include the impact of the ongoing voluntary liquidation of the Rare Diseases subsidiary in China, following the rejection of the National Reimbursement Drug List approval for Isturisa ® . The availability of Qarziba ® and Sylvant ® in the territory continues to be provided through a local distributor. EBITDA (1) was € 991.1 million for full year 2025, up 14.5% compared to full year 2024, with margin on net revenue of 37.8%. The improvement over the prior year was driven by a positive business mix and strong operating performance across both business units, despite the significant foreign exchange headwinds and higher investments to support the U.S. launch of the expanded Isturisa ® label, the continued development of Enjaymo ® and ongoing geographic expansion in Rare Diseases. Financial expenses were € 89.5 million for full year 2025, down by € 2.1 million as compared to full year 2024. New loans obtained in 2024, related to the acquisition of Enjaymo ® , and in 2025 led to an increase in interest expenses of € 17.4 million. Net exchange gains over the period were € 15.0 million (mainly unrealized and driven by the devaluation of the U.S. dollar), against net FX losses of € 9.3 million in FY 2024. This was partly offset by € 5.3 million of net monetary losses from hyperinflation accounting (compared to a loss of € 6.7 million in full year 2024) mainly driven by the net effect of the revaluation of Turkish balance sheet items. Adjusted Net Income was € 651.1 million, 24.9% of revenue, up by 14.5% compared to full year 2024. This growth reflects improvements in adjusted operating income as well as lower financial expenses partially offset by higher income taxes. Net income was € 443.6 million, 16.9% of revenue, increasing by 6.5% versus full year 2024, mainly driven by the higher operating income and lower financial expenses. Free cash flow (4) was € 558.8 million for full year 2025, an increase of € 23.7 million versus full year 2024, with strong EBITDA partially offset by higher working capital absorption (mainly driven by higher U.S. inventory levels), higher interests and income tax paid. Net debt (5) as of December 31, 2025 was € 2,037.3 million, or leverage of just below 2.1x EBITDA, compared to net debt of € 2,154.3 million on December 31, 2024, following dividend payments of € 267.6 million, treasury shares purchased for € 112.5 million (net of proceeds from exercising stock options), the upfront payment for Vazkepa ® rights of USD 25 million and the upfront payment for Inrebic ® rights of USD 11 million. Shareholders’ equity was € 1,919.8 million. Pipeline Update On April 15, 2025, the U.S. Food and Drug Administration (FDA) approved the supplemental new drug application (sNDA) for Isturisa ® (osilodrostat) for the treatment of endogenous hypercortisolemia in adults with Cushing’s syndrome for whom surgery is not an option or has not been curative. This was an expansion of the previous indication for the treatment of patients with Cushing’s disease, which is a sub-type of Cushing’s syndrome. The Isturisa ® indication expansion was supported by the extensive Isturisa ® clinical development program, which included over 350 patients. In addition, during the second quarter of 2025, Isturisa ® was granted regulatory approval in both Canada and Russia. A Phase IV study to assess the efficacy and safety of osilodrostat in adults with mild hypercortisolemia and uncontrolled hypertension (HTN) due to Cushing’s syndrome is expected to start in 2026. During the second quarter of 2025, an investigator-sponsored clinical trial (IST) was initiated to investigate the safety, dose and early signs of effect for dinutuximab beta (Qarziba ® ) in combination with chemotherapy for the treatment of patients with GD2-positive Ewing sarcoma. On July 28, 2025, the European Commission issued a positive decision and granted marketing authorization, under exceptional circumstances, for Maapliv ® , a solution of amino acids intended for the treatment of maple syrup urine disease (MSUD) presenting with an acute decompensation episode in patients from birth who are not eligible for an oral and enteral branched-chain amino acids (BCAA)-free formulation. The Company completed enrollment of the pasireotide Phase 2 trial for the treatment of post-bariatric hypoglycemia in August 2025. Top-line results are expected in the second quarter of 2026. Following the meeting with the U.S. Food and Drug Administration (FDA) in early September, a potential U.S. biologics license application (BLA) pathway was established with the FDA for Qarziba ® requiring an additional set of clinical data from the ongoing BEACON-2 investigator-sponsored trial. Results of the interim analysis are expected in the first half of 2028 and are expected to form the basis, together with existing clinical data, for a potential regulatory filing. On January 5, 2026, the UK Medicines and Healthcare products Regulatory Agency (MHRA) granted marketing authorization for Eligard ® for the treatment of hormone dependent advanced prostate cancer and for the treatment of high-risk localized and locally advanced hormone dependent prostate cancer in combination with radiotherapy. The other lifecycle management programs are progressing in line with plans. Corporate Development On June 24, 2025, Recordati announced a licensing and supply agreement with Amarin to commercialize the marketed cardiovascular medicine, Vazkepa ® (icosapent ethyl) across 59 countries, focused in Europe. Vazkepa ® is indicated to reduce the risk of cardiovascular events in statin-treated adult patients at high cardiovascular risk with elevated triglycerides and either established cardiovascular disease or diabetes with at least one other cardiovascular risk factor. Vazkepa ® is expected to achieve over € 40 million in revenues in 2027 and to be EBITDA positive from 2026. Under the terms of the agreement, Recordati paid Amarin an upfront cash payment of USD 25 million. On December 17, 2025, Recordati announced the exclusive license agreement with Impact Biomedicines, Inc., a Bristol Myers Squibb subsidiary, and the related supply agreement with Celgene Logistics Sàrl to commercialize Inrebic ® (fedratinib dihydrochloride monohydrate) in Japan. Impact Biomedicines, Inc. will retain exclusive rights to develop and commercialize Inrebic® in the rest of the world. Inrebic ® is an oral kinase inhibitor with activity against wild-type and mutationally activated JAK2 to suppress the pathological features of myelofibrosis patients. Inrebic ® received regulatory approval from the Ministry of Health, Labour and Welfare (MHLW) in Japan in June 2025 for the treatment of myelofibrosis and is expected to launch in mid 2026. Under the terms of the agreement, Recordati paid Impact Biomedicines, Inc. an upfront payment of USD 11 million. On January 29, 2026, Recordati announced a collaboration and license agreement with Moderna to develop and commercialize worldwide mRNA-3927, an investigational product for the treatment of propionic acidemia (PA). Under the terms of the agreement, Moderna will continue to lead the development of mRNA-3927, in collaboration with Recordati, and if approved, Recordati will lead global commercialization. mRNA-3927 is a post proof-of-concept, investigational product aimed to restore propionyl-CoA carboxylase (PCC) enzyme activity in patients with propionic acidemia. If approved, this could be the first disease-modifying treatment option on the market for this severe disease. mRNA-3927 is currently being evaluated in a potential registrational clinical study. The target patient enrollment has been reached, with a potential data readout expected by the end of 2026. Under the terms of the agreement, Recordati will pay Moderna an upfront payment of USD 50 million and up to an additional USD 110 million in near-term development and regulatory milestones. Moderna is also eligible to receive commercial and sales milestones, as well as tiered royalties on annual net sales. Recordati does not expect any significant impact on its EBITDA prior to a potential launch. Business outlook The financial targets for full year 2026 are as follows: Net revenue between € 2,730 and € 2,800 million with FX headwind of ~-3.5% EBITDA (1) between € 995 and € 1,030 million; margin of +/- 36.5% Adjusted net income (2) between € 655 and € 685 million; margin of +/- 24.0% The full year 2027 targets (9) remain unchanged, with strong organic growth complemented by bolt-on BD and M&A. (1) Net income before income taxes, financial income and expenses, depreciation, amortization and write-downs of property, plant and equipment, intangible assets and goodwill, non-recurring items and non-cash charges arising from the allocation of the purchase price of acquisitions to the gross margin of acquired inventory as foreseen by IFRS (2) Net income excluding amortization and write-downs of intangible assets (except software) and goodwill, non-recurring items, non-cash charges arising from the allocation of the purchase price of acquisitions to the gross margin of acquired inventory as foreseen by IFRS 3, monetary net gains/losses from hyperinflation (IAS 29), net of tax effects. (3) Proforma growth calculated excluding revenue of Vazkepa® for FY 2025 (Specialty & Primary Care) and Enjaymo® for both FY 2025 and FY 2024 (Rare Diseases) (4) Total cash flow excluding financing items, milestones, dividends, purchases of treasury shares net of proceeds from exercise of stock options. (5) Cash and cash equivalents, less bank debts and loans, which include the measurement at fair value of hedging derivatives. (6) Comparing FY 2025 revenue (which considers also the margin retained by Sanofi’s on in market sales for those countries where it was still holding the MA) with proforma FY 2024 revenue also including sales totally realized by Sanofi. (7) Net income before income taxes, financial income and expenses and non-recurring items, non-cash charges arising from the allocation of the purchase price of acquisitions to the gross margin of acquired inventory as foreseen by IFRS 3. (8) The provision has been revised since September to reflect the terms of the final settlement agreement with AIFA (9) FY 2027 targets: Net Revenue €3,000 - €3,200 million, EBITDA €1,140 - €1,225 million, Adjusted Net Income €770- €820 million, excluding potential impact from tariffs and/or most favored nation pricing policies in the U.S. Conference Call Recordati will host a conference call on February 18 th , at 2:00 p.m. CET ( 1:00 p.m. GMT ) to present the results for full year 2025. Please find the pre-registration link here with all the dial-in details and a calendar invitation to follow. Alternatively, if not pre-registered, the dial-in numbers for the conference call are: Italy + 39 02 802 09 11, toll free 800 231 525 UK + 44 1 212818004, toll free (44) 0 800 0156371 USA +1 718 7058796, toll free (1) 1 855 2656958 France +33 1 70918704 Germany +49 6917415712 Participants are invited to dial in 10 minutes before the start of the conference call. If operator assistance is required to connect, please dial *0. The slides that will be referenced during the call will be available at under Investors/Company Presentations. Recordati is an international pharmaceutical group listed on the Italian Stock Exchange (XMIL: REC), with roots dating back to a family-run pharmacy in Northern Italy in the 1920s. We are uniquely structured to provide treatments across specialty and primary care, and rare diseases. Our fully integrated operations span clinical development, chemical and finished product manufacturing, commercialization and licensing. We operate in approximately 150 countries across EMEA, the Americas and APAC with over 4,500 employees. We believe that health is a fundamental right, not a privilege. Today, our purpose of “unlocking the full potential of life” aims at empowering individuals to live life to the fullest, whether addressing common health challenges or the rarest. Investor Relations Eugenia Litz eugenia.litz@recordati.com Gianluca Saletta saletta.g@recordati.it Media Relations Laura Conti conti.l@recordati.it Rebecca Kerr rebecca.kerr@recordati.com This document contains forward-looking statements relating to future events and future operating, economic and financial results of the Recordati group. By their nature, forward-looking statements involve risk and uncertainty because they depend on the occurrence of future events and circumstances. Actual results may therefore differ materially from forecasts for a variety of reasons, most of which are beyond the Recordati group ’ s control. The information on the pharmaceutical specialties and other products of the Recordati group contained in this document is intended solely as information on the activities of the Recordati Group, and, as such, it is not intended as a medical scientific indication or recommendation, or as advertising . DECLARATION BY THE FINANCIAL REPORTING OFFICER The Financial Reporting Officer, Niccolò Giovannini, declares, pursuant to paragraph 2 of Article 154- bis of the Consolidated Law on Finance, that the accounting information contained in this press release corresponds to the Company’s documentation, books and accounting records. RECORDATI INDUSTRIA CHIMICA E FARMACEUTICA S.p.A. Registered Office Via Matteo Civitali, 1 20148 Milano, Italy Tel. +39 02 487871 Fax +39 02 40073747 Share Capital € 26.140.644,50 fully paid-up Milano, Monza, Brianza and Lodi Comp. Reg. No. 00748210150 Tax Code/VAT No. 00748210150 Milano R.E.A. No. 401832 Company subject to the Management and Coordination Activity of Rossini Luxembourg S.àr.l

License out/inDrug ApprovalPhase 2Financial Statement

03 Sep 2025

·www.prnewswire.com

Myelofibrosis Market is Going to Grow at a CAGR of 9% in the Next Ten Years (2025-2034) | DelveInsight

The launch of emerging therapies like BESREMi (PharmaEssentia and AOP Orphan Pharmaceuticals), INCB057643 (Incyte), XPOVIO (Karyopharm Therapeutics), RYTELO (Geron), REBLOZYL (Bristol Myers Squibb), Navtemadlin (Kartos Therapeutics), Pelabresib (Novartis), and others is going to shift the myelofibrosis market. LAS VEGAS, Sept. 3, 2025 /PRNewswire/ -- DelveInsight's Myelofibrosis Market Insights report includes a comprehensive understanding of current treatment practices, emerging myelofibrosis drugs, market share of individual therapies, and current and forecasted myelofibrosis market size from 2020 to 2034, segmented into leading markets (the US, EU4, UK, and Japan). Myelofibrosis Market Summary The total myelofibrosis treatment market size in the leading markets (the US, EU4, UK, and Japan) was USD 2.2 billion in 2024. The United States accounts for the largest market size of myelofibrosis, in comparison to EU4 (Germany, Italy, France, and Spain) and the UK, and Japan. Based on DelveInsight's assessment in 2024, the 7MM had approximately 56K patient pool (prevalent cases) of myelofibrosis. Key myelofibrosis companies, including PharmaEssentia, AOP Orphan Pharmaceuticals, Incyte, Karyopharm Therapeutics, Geron, Bristol Myers Squibb, Kartos Therapeutics, Novartis, Merck, Telios Pharma, Ryvu Therapeutics, Sumitomo Pharma, Syntara, Disc Medicine, Menarini Group, and others, are actively working on innovative myelofibrosis drugs. Some of the key myelofibrosis therapies in clinical trials include BESREMi (ropeginterferon alfa-2b-njft/P-1101), INCB057643, XPOVIO (NEXPOVIO/selinexor/KPT-330), RYTELO (imetelstat), REBLOZYL (luspatercept/ACE-536), Navtemadlin (KRT-232), Pelabresib (DAK539), Bomedemstat (IMG-7289/MK-3543), TL-895, RVU120 ( SEL-120), TP-3654 (nuvisertib), SNT-5505 (PXS-5505), DISC-0974, ELZONRIS (tagraxofusp/SL-401), and others. These novel myelofibrosis therapies are anticipated to enter the myelofibrosis market in the forecast period and are expected to change the market. By 2034, among all the therapies, the highest revenue is expected to be generated by OJJAARA/OMJJARA. Discover which myelofibrosis therapies are expected to grab the largest market share @ Myelofibrosis Market Report Key Factors Driving the Growth of the Myelofibrosis Market Novel Therapies Drive Myelofibrosis Treatment Advancements Currently, four JAK inhibitors have been approved by the US FDA for the treatment of myelofibrosis, including JAKAFI (ruxolitinib), INREBIC (fedratinib), VONJO (pacritinib), and OJJAARA (momelotinib). No drug therapy can cure myelofibrosis. As myelofibrosis primarily affects older adults, stem cell transplantation is not a treatment option for most myelofibrosis patients. The launch of another JAK inhibitor, BESREMi, will further change the dynamics of the myelofibrosis market. Rich clinical pipeline and emerging non-JAK approaches driving the myelofibrosis landscape Beyond JAK inhibitors, active clinical development includes BET inhibitors (Incyte's INCB057643; Novartis' Pelabresib), XPO1 inhibitor (Karyopharm Therapeutics' XPOVIO), Telomerase inhibitor (Geron's RYTELO), MDM2 protein inhibitor (Kartos Therapeutics' Navtemadlin), Tyrosine kinase inhibitors (Telios Pharma's TL-895), PIM1 kinase inhibitor (Sumitomo Pharma's TP-3654), LOX inhibitor (Syntara's SNT-5505), and others, raising market potential by promising new label expansions, second-line options, and higher-value therapies should any prove to be disease-modifying. An aging population & increasing incidence with better survival Myelofibrosis primarily affects older adults; population aging increases the absolute number of patients. As treatments improve symptoms and (in some cases) survival, prevalent patient pools grow, supporting longer-term market demand. In the US in 2024, the 70+ years of age group had the highest number of cases, accounting for approximately 60% of the total prevalent cases, while the ≤39 years of age group accounted for just ~2%. Diagnostic & genomic testing improvements are driving myelofibrosis patient pool, leading to the growth of myelofibrosis market Wider use of molecular profiling (JAK2, CALR, MPL, and others) enables earlier and more accurate MF diagnosis and better patient stratification for targeted therapies. This both expands the diagnosed population and helps match patients to appropriate, often higher-value, treatments. Myelofibrosis Market Analysis JAK inhibitors have become the cornerstone of treatment for patients with myelofibrosis, offering significant benefits such as spleen reduction, symptom relief, and improved quality of life, which can also extend survival in those with advanced disease. All approved JAK inhibitors primarily target JAK2, particularly the wild-type form, but they differ in their activity against other JAK family members. For instance, JAKAFI myelofibrosis inhibits both JAK1 and JAK2; INREBIC selectively inhibits JAK2 while sparing JAK1 and also affects FLT3 and other targets; VONJO myelofibrosis inhibits JAK2 while sparing JAK1 but additionally impacts FLT3, IRAK, and ACVR1; and OJJAARA, approved through a different pathway, targets JAK1/JAK2 and ACVR1, mainly for myelofibrosis patients with anemia. These varying mechanisms lead to distinct patient outcomes. JAKAFI myelofibrosis continues to see strong demand and is expected to grow further, maintaining its position as the standard of care in myelofibrosis. Myelofibrosis will remain the largest segment of JAKAFI's patient population until polycythemia vera cases eventually increase. However, market growth may be limited due to patent expirations of key therapies, with JAKAFI patents set to expire in 2027 for Novartis and 2028 for Incyte, presenting potential opportunities for competitors. In response, Incyte is exploring combination trials with novel drugs to extend JAKAFI's therapeutic lifespan. Learn more about the treatment options for myelofibrosis @ Myelofibrosis Therapy The myelofibrosis treatment landscape offers significant opportunities for innovation, focusing on several key areas: Treating Lower-Risk Patients: Developing safe and effective therapies for patients with lower-risk myelofibrosis, allowing for earlier and more favorable interventions. Enhancing First-Line Treatments: Improving existing first-line therapies for intermediate- to high-risk patients through novel drugs or combination strategies. Managing Cytopenia: Developing targeted therapies to address cytopenia, a significant unmet need in patient care. Myelofibrosis Competitive Landscape The myelofibrosis clinical trial pipeline includes several drugs in mid- and late-stage development that are expected to enter the market during the forecast period. The emerging landscape offers a diverse range of therapeutic alternatives for treatment, including XPOVIO (Karyopharm Therapeutics), Elitracet (Takeda/Keros Therapeutics), Navtemadlin (Kartos Therapeutics), Pelabresib (Novartis), INCB057643 (Incyte), Bomedemstat (Merck), and others, all of which are used in various lines of treatment. The expected launch of these therapies is expected to have a further positive impact on the market. INCB57643 is an orally administered small molecule. Bromodomain and extra-terminal (BET) proteins act as epigenetic readers that control the expression of key oncoproteins implicated in the development of myelofibrosis and other hematologic malignancies, including B-lymphoma-2, nuclear factor kappa, and c-Myc. In a prior Phase I/II clinical trial, the oral BET inhibitor INCB057643, tested both as a monotherapy and in combination with ruxolitinib, demonstrated favorable tolerability and promising clinical activity in patients with advanced cancers. In its Q2 2025 financial report, the company announced that the combination of INCB057643 with ruxolitinib and INCB57643 (a JAK1/JAK2 and BET inhibitor) is currently being evaluated in a Phase II trial for myelofibrosis. RYTELO (imetelstat) is an investigational telomerase inhibitor that targets telomerase, selectively eliminating malignant stem and progenitor cells in the bone marrow, which drive diseases like myelodysplastic syndromes (MDS) and myelofibrosis. By blocking the proliferation of these malignant cells, imetelstat supports the recovery of healthy bone marrow and blood cell production and has shown disease-modifying effects and clinical benefits in Phase III trials for myelofibrosis. This mechanism sets imetelstat apart from other approved or investigational therapies for these blood cancers. In January 2025, Geron reported achieving 75% enrollment in the Phase III IMpactMF trial, which is comparing imetelstat to Best Available Therapy (BAT) in intermediate-2 or high-risk myelofibrosis patients who have relapsed or are refractory to JAK inhibitor treatment. Elritercept is a late-stage investigational activin inhibitor aimed at treating anemia associated with hematologic malignancies, including MDS and myelofibrosis. It is currently undergoing Phase II evaluation in patients with myelofibrosis. In December 2024, Keros Therapeutics presented updated data from this ongoing Phase II trial at ASH 2024. Additionally, in December 2024, Takeda announced an exclusive licensing agreement with Keros to further develop, manufacture, and commercialize elritercept globally, excluding mainland China, Hong Kong, and Macau. The anticipated launch of these emerging myelofibrosis therapies are poised to transform the myelofibrosis market landscape in the coming years. As these cutting-edge myelofibrosis therapies continue to mature and gain regulatory approval, they are expected to reshape the myelofibrosis market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth. To know more about new myelofibrosis treatment, visit @ Myelofibrosis Treatment Market Recent Developments in the Myelofibrosis Market In July 2025, Incyte announced that the Phase I data in patients with myelofibrosis as monotherapy and in combination with ruxolitinib are anticipated in the second half of 2025. In June 2025, QIAGEN and Incyte announced a new global collaboration to develop a novel diagnostic panel to support Incyte's extensive portfolio of investigational therapies for patients with myeloproliferative neoplasms (MPNs), including Incyte's monoclonal antibody INCA033989. In January 2025, Karyopharm Therapeutics stated that it expects to report top-line results from the Phase III SENTRY trial in the second half of 2025, which could represent a potentially transformative opportunity to establish a new treatment paradigm in myelofibrosis. What is Myelofibrosis? Myelofibrosis is a rare blood cancer marked by the accumulation of scar tissue, or 'fibrosis', in the bone marrow. This excess scar tissue prevents the bone marrow from producing enough healthy blood cells. It belongs to a group of blood cancers called 'myeloproliferative neoplasms (MPNs)', in which the blood cells produced by the bone marrow grow and function abnormally. When myelofibrosis arises independently, without being caused by another bone marrow disorder, it is referred to as primary myelofibrosis. In other cases, it can develop from another MPN, such as polycythemia vera or essential thrombocythemia. When this occurs, it is called secondary myelofibrosis, sometimes specifically termed post-polycythemia vera myelofibrosis or post-essential thrombocythemia myelofibrosis. Myelofibrosis Epidemiology Segmentation The myelofibrosis epidemiology section provides insights into the historical and current myelofibrosis patient pool and forecasted trends for the leading markets (the US, EU4, UK, and Japan). It helps recognize the causes of current and forecasted patient trends by exploring numerous studies and views of key opinion leaders. The myelofibrosis market report proffers epidemiological analysis for the study period 2020–2034 in the leading markets (the US, EU4, UK, and Japan) segmented into: Total Prevalent Cases of Myelofibrosis Type-specific Cases of Myelofibrosis Myelofibrosis Cases Based on Risk Stratification Age-specific Prevalent Cases of Myelofibrosis Myelofibrosis Cases Based on Molecular Alterations Download the report to understand which factors are driving myelofibrosis epidemiology trends @ Myelofibrosis Treatment Drugs Scope of the Myelofibrosis Market Report Myelofibrosis Therapeutic Assessment: Myelofibrosis current marketed and emerging therapies Myelofibrosis Market Dynamics: Conjoint Analysis of Emerging Myelofibrosis Drugs Competitive Intelligence Analysis: SWOT analysis and Market entry strategies Myelofibrosis Market Unmet Needs, KOL's views, Analyst's views, Myelofibrosis Market Access and Reimbursement Discover more about myelofibrosis drugs in development @ Myelofibrosis Clinical Trials Table of Contents Related Reports JAK Inhibitors Market JAK Inhibitors Market Size, Target Population, Competitive Landscape, and Market Forecast – 2034 report delivers an in-depth understanding of the market trends, market drivers, market barriers, and key JAK inhibitors companies, including Pfizer, AbbVie, Galapagos, Sierra Oncology, Theravance Biopharma, Dizal Pharmaceutical, Aclaris Therapeutics, Celon Pharma, Incyte Corporation, Gilead Sciences, Reistone Biopharma, Jiangsu Hengrui Medicine Co., MaxiNovel Pharmaceuticals, among others. Myelofibrosis Clinical Trial Analysis Myelofibrosis Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key myelofibrosis companies, including Geron Corporation, Merck, Ryvu Therapeutics SA, Morphic Therapeutic, iOnctura, Pharmaxis, Nippon Shinyaku, Active Biotech, Incyte Corporation, Sumitomo Pharma America, Inc., Cellenkos, Disc Medicine, among others. Polycythemia Vera Market Polycythemia Vera Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key polycythemia vera companies, including Incyte, Novartis, PharmaEssentia, AOP Orphan Pharmaceuticals, Protagonist Therapeutics, Merck (Imago BioSciences), Italfarmaco, Ionis Pharmaceutical, Silence Therapeutics, Perseus Proteomics, AbbVie, Johnson & Johnson Innovative Medicine, Mabwell (Shanghai) Bioscience, Disc Medicine, GluBio Therapeutics, among others. Multiple Myeloma Market Multiple Myeloma Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key multiple myeloma companies, including Sanofi, Karyopharm Therapeutics, AbbVie, Takeda Pharmaceutical, Celgene, Bristol-Myers Squibb, RAPA Therapeutics, Pfizer, Array Biopharma, Cellectar Biosciences, BioLineRx, Celgene, Aduro Biotech, ExCellThera, Janssen Pharmaceutical, Precision BioSciences, Takeda, Glenmark (Ichnos Sciences SA), Poseida Therapeutics, Molecular Partners AG, Chipscreen Biosciences, AbbVie, Genentech (Roche), Janssen Biotech, Nanjing Legend Biotech, Merck Sharp & Dohme Corp., among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Connect with us on LinkedIn | Facebook | Twitter Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Phase 2License out/inPhase 1Drug ApprovalClinical Result

28 Aug 2025

·www.prnewswire.com

Vanda Pharmaceuticals Announces FDA Granted Orphan Drug Designation for VGT-1849B, a Novel and Selective Candidate for the Treatment of Polycythemia Vera

WASHINGTON, Aug. 28, 2025 /PRNewswire/ -- Vanda Pharmaceuticals Inc. (Vanda) (Nasdaq: VNDA) today announced the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for VGT-1849B, a selective peptide nucleic acid-based JAK2 inhibitor for the treatment of polycythemia vera (PV). PV is a chronic myeloproliferative disorder characterized by aberrant hematopoiesis of myeloid lineage with exuberant red cell production and increased release of pro-inflammatory cytokines. More than 95% of PV patients harbor the JAK2 V617F gain-of-function mutation leading to aberrant JAK2 production.1 The prevalence of PV in the United States is estimated to affect 44 to 57 per 100,000 people.2 VGT-1849B is an antisense oligonucleotide (ASO) that utilizes a novel backbone chemistry, OliPass Peptide Nucleic Acid (OPNA), that has been derived from peptide nucleic acid (PNA) by rationally introducing cationic lipid moieties onto nucleobases. By covalently attaching cationic lipid groups onto PNA, cell permeability and affinity for RNA are markedly improved. By selectively targeting JAK2 mRNA, VGT-1849B reduces downstream signaling and JAK2V617F-driven autonomous cell proliferation. VGT-1849B targets JAK2 with high precision and effectively reduces JAK2 protein production, without any off-target kinase effects. Inhibiting JAK2 acts to suppress hematopoiesis, consequently reducing red blood cell, neutrophil, platelet, and lymphocyte production. The ability of VGT-1849B to reduce JAK2 protein may alleviate the disease burden that patients with PV face with a favorable safety profile, resulting in a higher quality of life for patients. JAK2 inhibitors have been shown to be efficacious in treating various JAK-dependent hematologic malignancies, including the treatment of PV. While there are several JAK inhibitors available such as Jakafi®, Inrebic®, Ojjaara®, and Vonjo®, none are solely selective to JAK2. Due to the highly conserved structure of the catalytic sites of protein kinases, JAK2 inhibitors may bind to off-target kinases, leading to increased toxicity and a worse overall safety profile. The adverse side effects that may occur from JAK inhibition emphasize the importance of selectively targeting JAK2 while avoiding inhibition of other JAK family members. By specifically targeting JAK2, we aim to reduce the risk of infection and toxic effects that are seen with inhibitors that also block JAK1, JAK3, TYK2, or other kinases outside of the JAK family. If approved, VGT-1849B could offer targeted efficacy with an improved safety profile and convenient infrequent dosing. Orphan Drug Designation is granted by the FDA to investigational therapies addressing rare medical conditions and provides benefits to drug developers. References P. Gou, W. Zhang, and S. Giraudier, "Insights into the Potential Mechanisms of JAK2V617F Somatic Mutation Contributing Distinct Phenotypes in Myeloproliferative Neoplasms," Myeloproliferative Neoplasms. Int. J. Mol. Sci, vol. 2022, p. 1013, 2022, doi: 10.3390/ijms. Grunwald, M. R.; Stein, B. L.; Boccia, R. V.; Oh, S. T.; Paranagama, D.; Parasuraman, S.; Colucci, P.; Mesa, R. Clinical and Disease Characteristics From REVEAL at Time of Enrollment (Baseline): Prospective Observational Study of Patients With Polycythemia Vera in the United States. Clin Lymphoma Myeloma Leuk 2018, 18 (12), 788-795.e2. . About Vanda Pharmaceuticals Inc. Vanda is a leading global biopharmaceutical company focused on the development and commercialization of innovative therapies to address high unmet medical needs and improve the lives of patients. For more on Vanda Pharmaceuticals Inc., please visit and follow us on X @vandapharma. About VGT-1849B VGT-1849B is an antisense oligonucleotide (ASO) that utilizes a novel backbone chemistry, OliPass Peptide Nucleic Acid (OPNA) peptide nucleic acid, and selectively targets JAK2 mRNA, reducing aberrant levels of JAK2 that may cause hematologic malignancies. OPNA was derived from PNA through rational chemical modifications to enhance cell permeability and RNA affinity. For therapeutic intervention, OPNA potently binds to target pre-mRNA, induces exon skipping, and yields an mRNA splice variant. Unlike other types of RNA therapeutics, OPNA does not require formulation aid for in vivo therapeutic activity. OPNA oligonucleotide-based therapeutics have broad applicability in addressing a number of disorders caused by genetic variants. CAUTIONARY NOTE REGARDING FORWARD-LOOKING STATEMENTS Various statements in this press release, including, but not limited to statements regarding the estimated prevalence of PV in the United States, the potential therapeutic effects of VGT-1849B, the safety profile of VGT-1849B and the potential benefits of VGT-1849B are "forward-looking statements" under the securities laws. All statements other than statements of historical fact are statements that could be deemed forward-looking statements. Forward-looking statements are based upon current expectations and assumptions that involve risks, changes in circumstances and uncertainties. Important factors that could cause actual results to differ materially from those reflected in Vanda's forward-looking statements include, among others, the accuracy of the reporting and diagnosis of PV cases, the ability of VGT-1849B to safely and effectively treat PV, the ability of VGT-1849B to provide patients with the anticipated benefits and Vanda's ability to successfully complete the clinical development of, and obtain regulatory approval for, VGT-1849B in the treatment of PV. Therefore, no assurance can be given that the results or developments anticipated by Vanda will be realized or, even if substantially realized, that they will have the expected consequences to, or effects on, Vanda. Forward-looking statements in this press release should be evaluated together with the various risks and uncertainties that affect Vanda's business and market, particularly those identified in the "Cautionary Note Regarding Forward-Looking Statements", "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations" sections of Vanda's most recent Annual Report on Form 10-K, as updated by Vanda's subsequent Quarterly Reports on Form 10-Q, Current Reports on Form 8-K and other filings with the U.S. Securities and Exchange Commission, which are available at . All written and verbal forward-looking statements attributable to Vanda or any person acting on its behalf are expressly qualified in their entirety by the cautionary statements contained or referred to herein. Vanda cautions investors not to rely too heavily on the forward-looking statements Vanda makes or that are made on its behalf. The information in this press release is provided only as of the date of this press release, and Vanda undertakes no obligation, and specifically declines any obligation, to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law. Corporate Contact: Kevin Moran Senior Vice President, Chief Financial Officer and Treasurer Vanda Pharmaceuticals Inc. 202-734-3400 [email protected] Jim Golden / Jack Kelleher / Dan Moore Collected Strategies [email protected] SOURCE Vanda Pharmaceuticals Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Orphan DrugOligonucleotide

100 Deals associated with Fedratinib Hydrochloride

External Link

KEGG	Wiki	ATC	Drug Bank
-	Fedratinib Hydrochloride	L01XE57	DB12500

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Post-essential thrombocythemia myelofibrosis	European Union	Bristol-Myers Squibb Pharma EEIG	08 Feb 2021
Post-essential thrombocythemia myelofibrosis	Iceland	Bristol-Myers Squibb Pharma EEIG	08 Feb 2021
Post-essential thrombocythemia myelofibrosis	Liechtenstein	Bristol-Myers Squibb Pharma EEIG	08 Feb 2021
Post-essential thrombocythemia myelofibrosis	Norway	Bristol-Myers Squibb Pharma EEIG	08 Feb 2021
Post-polycythemia vera myelofibrosis	European Union	Bristol-Myers Squibb Pharma EEIG	08 Feb 2021
Post-polycythemia vera myelofibrosis	Iceland	Bristol-Myers Squibb Pharma EEIG	08 Feb 2021
Post-polycythemia vera myelofibrosis	Liechtenstein	Bristol-Myers Squibb Pharma EEIG	08 Feb 2021
Post-polycythemia vera myelofibrosis	Norway	Bristol-Myers Squibb Pharma EEIG	08 Feb 2021
Primary Myelofibrosis	European Union	Bristol-Myers Squibb Pharma EEIG	08 Feb 2021
Primary Myelofibrosis	Iceland	Bristol-Myers Squibb Pharma EEIG	08 Feb 2021
Primary Myelofibrosis	Liechtenstein	Bristol-Myers Squibb Pharma EEIG	08 Feb 2021
Primary Myelofibrosis	Norway	Bristol-Myers Squibb Pharma EEIG	08 Feb 2021
Splenomegaly	European Union	Bristol-Myers Squibb Pharma EEIG	08 Feb 2021
Splenomegaly	Iceland	Bristol-Myers Squibb Pharma EEIG	08 Feb 2021
Splenomegaly	Liechtenstein	Bristol-Myers Squibb Pharma EEIG	08 Feb 2021
Splenomegaly	Norway	Bristol-Myers Squibb Pharma EEIG	08 Feb 2021
Myelofibrosis	United States	Bristol Myers Squibb Co.	16 Aug 2019
Polycythemia Vera	United States	Bristol Myers Squibb Co.	16 Aug 2019
Thrombocythemia, Essential	United States	Bristol Myers Squibb Co.	16 Aug 2019

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Thrombocytopenia	Phase 3	China	Celgene Corp.	05 Aug 2019
Thrombocytopenia	Phase 3	Australia	Celgene Corp.	05 Aug 2019
Thrombocytopenia	Phase 3	Austria	Celgene Corp.	05 Aug 2019
Thrombocytopenia	Phase 3	Belgium	Celgene Corp.	05 Aug 2019
Thrombocytopenia	Phase 3	Czechia	Celgene Corp.	05 Aug 2019
Thrombocytopenia	Phase 3	France	Celgene Corp.	05 Aug 2019
Thrombocytopenia	Phase 3	Germany	Celgene Corp.	05 Aug 2019
Thrombocytopenia	Phase 3	Hungary	Celgene Corp.	05 Aug 2019
Thrombocytopenia	Phase 3	Ireland	Celgene Corp.	05 Aug 2019
Thrombocytopenia	Phase 3	Italy	Celgene Corp.	05 Aug 2019

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


ISRCTN88102629 (FEDORA, ASH2025)	Phase 2	Myelofibrosis JAK2V617F positive	34	Fedratinib 400mg + Ropeginterferon Alfa-2b	yvqogjyjvb(xwvdmpujng) = kajvcasugb hgiulhmbxi (mcofbphobp ) View more	Positive	06 Dec 2025
FRACTION (ASH2025)	Phase 2	Myelofibrosis	30	FedratinibNivolumabb + FedratinibNivolumab	szyamkaghm(xnvqdczxca) = scigyiqsjy gqacoqdglb (ewyygzofim ) View more	Positive	06 Dec 2025
ASCO2025	Not Applicable	Myelofibrosis JAK2 \| FLT3	960	Fedratinib after Ruxolitinib	czoqvzpgpt(pugtnlwxhr) = 31.2% (C.I. 0.27-0.36, p < 0.001) hhwygfaast (zhmmyvhatk ) View more	Positive	30 May 2025
				Ruxolitinib alone
NCT03952039 (FREEDOM2, Pubmed \| Lancet Haematol) Manual	Phase 3	Myelofibrosis Second line	201	Fedratinib 400 mg per day	zeeprqtxse(tmahbjfsux) = yaycpbvvaa ihhwmlgmuf (vserwzhpwb ) View more	Positive	01 Sep 2024
				Best Available Therapy (BAT)	zeeprqtxse(tmahbjfsux) = yvxvmzxanu ihhwmlgmuf (vserwzhpwb ) View more
NCT03755518 (FREEDOM, Pubmed \| Leuk Lymphoma)	Phase 3	Myelofibrosis	38	Fedratinib	fiblxjiqhg(lfzmlwgzzs) = tsjmyfnlok idfsdplvjx (ejgqvdpcpa, 12.5 - 43.3) View more	Positive	28 Jul 2024
NCT03952039 (FREEDOM2, EHA2024) Manual	Phase 3	Myelofibrosis Second line	201	Fedratinib 400mg daily	jfygsyeckz(ibpitfljwk) = oliaxodzaa hjrzwfjeoz (npxocbhwbk ) View more	Positive	14 May 2024
				Best Available Therapy (BAT)	jfygsyeckz(ibpitfljwk) = erxnvqedoi hjrzwfjeoz (npxocbhwbk ) View more
Tandem Meetings ASTCT and CIBMTR2024	Not Applicable	Myelofibrosis	18	Fedratinib 400 mg once daily	qdiraqpmwm(zlndiuxzqn) = n=4 (22.2%) deaths were recorded as of May 2023. Two of the 4 deaths were related to MF and both patients had disease transformation to blast phase MPN before HSCT. A median duration of 14.5 months elapsed between FEDR initiation and death among these 4 patients ocefqwhnxc (lgxgzaqrnl ) View more	Positive	01 Feb 2024
NCT03952039 (FREEDOM2, CTgov)	Phase 3	Post-essential thrombocythemia myelofibrosis \| Post-polycythemia vera myelofibrosis \| Primary Myelofibrosis	202	Best Available Therapy (BAT)	dmfoolhpkw = xfrbwgfjqc ebxtaartxf (eeglquopjg, rdsvirnidf - jelevxikrl) View more	-	30 Jan 2024
NCT03952039 (FREEDOM2, ASH2023)	Phase 3	Myelofibrosis	201	Fedratinib 400 mg/day	wppkhwbwlu(lqubnyoyql) = khjbhknftf pmqprygxdf (dmsralvykr ) View more	Positive	10 Dec 2023
				Best Available Therapy (BAT)	wppkhwbwlu(lqubnyoyql) = sxyvgtlnsx pmqprygxdf (dmsralvykr ) View more
NCT04817007 (CA011-023, ASH2023)	Phase 1/2	Primary Myelofibrosis	40	BMS-986158+Ruxolitinib	jpuiinqxoz(bzdkuiwzap) = gpfdxacing jwflqagnwr (hgummpmkwe )	-	10 Dec 2023
				BMS-986158+Fedratinib	jpuiinqxoz(bzdkuiwzap) = clbveushxf jwflqagnwr (hgummpmkwe )

Translational Medicine

Boost your research with our translational medicine data.

view full example data

Deal

Boost your decision using our deal data.

view full example data

Core Patent

Boost your research with our Core Patent data.

view full example data

Clinical Trial

Identify the latest clinical trials across global registries.

view full example data

Approval

Accelerate your research with the latest regulatory approval information.

view full example data

Regulation

Understand key drug designations in just a few clicks with Synapse.

view full example data

AI Agents Built for Biopharma Breakthroughs

Accelerate discovery. Empower decisions. Transform outcomes.

Get started for free today!

Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.

Start your data trial now!

Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.

Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis