FGFR1 antagonists(Fibroblast growth factor receptor 1 antagonists), FGFR2 antagonists(Fibroblast growth factor receptor 2 antagonists), FGFR3 antagonists(Fibroblast growth factor receptor 3 antagonists)

Therapeutic Areas

NeoplasmsCongenital DisordersDigestive System Disorders+ [7]

Active Indication

Advanced Bile Duct CarcinomaChromosome 8p11 myeloproliferative syndromeBile Duct Neoplasms+ [19]

Inactive Indication

Advanced cancerBreast CancerChromosomal translocation+ [9]

Originator Organization

Incyte Corp.

Active Organization

Incyte Corp.Incyte Biosciences Distribution BV

Handok, Inc.

+ [4]

Inactive Organization-

Drug Highest PhaseApproved

First Approval Date

United States (17 Apr 2020),

FGFR2 positive Intrahepatic Cholangiocarcinoma

RegulationAccelerated Approval (United States), Orphan Drug (United States), Orphan Drug (European Union), Priority Review (China), Conditional marketing approval (China), Orphan Drug (South Korea), Orphan Drug (Australia), Priority Review (United States), Breakthrough Therapy (United States)

Structure/Sequence

Molecular FormulaC24H27F2N5O4

InChIKeyHCDMJFOHIXMBOV-UHFFFAOYSA-N

CAS Registry1513857-77-6

Clinical Trials associated with Pemigatinib

NCT06906562

/ Not yet recruitingPhase 2IIT

A Phase II Telemedicine Study of Pemigatinib in Adult Patients With Advanced or Metastatic Pancreas Cancer With FGFR2 Gene Fusions or Other FGFR Genetic Alterations

This study is designed to evaluate the efficacy of the targeted, selective panFGFR inhibitor Pemigatinib when administered as a single agent to patients with genetically selected advanced or metastatic pancreas cancer through assessment of the overall response rate. Additionally, this study will support the molecular characterization of this subset of pancreas cancer through correlative studies

Start Date01 May 2025

Sponsor / Collaborator

Ohio State University Comprehensive Cancer Center

[+1]

NCT06653777

/ Not yet recruitingPhase 2IIT

Phase II Trial Evaluating the Efficacy of Pemigatinib in Patients With Recurrent and/or Metastatic Solid Tumor Harboring a FGFR Alteration

Alterations in the fibroblast growth factor receptor (FGFR) gene are involved in the development of cancer. These anomalies are found at very variable frequencies (from less than 1% to around 10%) in cancers of the bile ducts, bladder, uterus, brain, ovary, lung, airways, digestive tract, breast, etc.
Pemigatinib is an anti-cancer drug that acts on cells with alterations in the FGFR gene. It is used in Europe to treat people with biliary tract cancer who carry a specific FGFR alteration.
However, in various clinical trials, pemigatinib has shown interesting activity in a number of patients with different cancers presenting with an alteration in the FGFR gene. This treatment could therefore be effective in several types of cancer where an alteration in the FGFR gene is detected.
The aim of this clinical trial is to learn if pemigatinib works to treat patients with recurrent and/or metastatic cancer (whatever the type of cancer excluding blood cancers and those already treated with pemigatinib) presenting an alteration in the FGFR gene.
Patients will:
* Take oral pemigatinig in 3-week cycles (every day for 2 weeks followed by one week without pemigatinib) as long as they benefit from it.
* Visit the clinic once every 3 weeks for checkups and tests during the treatment period
* Visit the clinic once every 3 months for checkups after stopping treatment for at least 12 months.

Start Date01 Apr 2025

Sponsor / Collaborator

UNICANCER

[+2]

NCT06728410

/ Not yet recruitingPhase 2IIT

A Phase II Study of Pemigatinib Plus Durvalumab (MEDI4736) in Previously Treated Advanced Intrahepatic Cholangiocarcinoma Patients with FGFR-2 Fusion or Rearrangement

This is a single arm phase II study of pemigatinib and durvalumab combination in patients with FGFR-2 fusion or rearrangement positive intrahepatic cholangiocarcinoma. Each cycle will be 3 weeks. Pemigatinib is administered at 13.5 mg orally daily 2 weeks on and 1 week off. Durvalumab is administered at 1500 mg intravenously once every 3 weeks. Subjects will require a visit with appropriate laboratory work prior to the start of each cycle. Disease assessment will occur every 9 weeks. Subjects will continue treatment until progression per RECIST 1.1, toxicity or subject/physician decision. A maximum of 24 months (about 35 cycles) of pemigatinib and durvalumab treatment from Cycle 1 Day 1 is allowed.

Start Date01 Mar 2025

Sponsor / Collaborator

Hoosier Cancer Research Network

[+3]

100 Clinical Results associated with Pemigatinib

100 Translational Medicine associated with Pemigatinib

100 Patents (Medical) associated with Pemigatinib

282

Literatures (Medical) associated with Pemigatinib

01 Jun 2025·ANALYTICAL BIOCHEMISTRY

A sensitive HPLC method with fluorescence detection for quantification of pemigatinib in human plasma samples and its in-vivo application to pharmacokinetic study in rats

Article

Author: Darwish, Ibrahim A ; Alzoman, Nourah Z ; Alrubia, Sarah ; Alsegiani, Amsha S

Cholangiocarcinoma is a lethal tumour of the bile ducts. Cholangiocarcinoma fibroblast growth factor receptor gene fusions forms can be targeted by Pemigatinib (PGT). PGT a recently approved kinase inhibitor by the US-FDA, has its approval accelerated due to the disease viciousness. Development of a sensitive yet available and economical analytical platform to quantify PGT in human plasma is genuinely needed. Enabling monitoring of the therapeutic plan, hence, ensuring the drug efficacy and safety through pharmacokinetic studies. High-performance liquid chromatography with fluorescence detector (HPLC-FD) method is proposed using the native fluorescence of PGT. PGT and seliciclib (internal standard) chromatographic conditions optimisation, revealed favourable use of isocratic mobile phase consisting of methanol:ammonium acetate buffer (70:30v/v, pH5.0) pumped into C18-column (150 mm length × 4.6 mm internal diameter, 5 μm particle size), at 1 mL min^-1 flow rate. PGT and seliciclib fluorescence excitation and emission were measured at 280 and 360 nm, respectively. Validation of the HPLC-FD method was processed based on the International Council for Harmonization guidelines. The method linearity range was 5-300 ng mL^-1. The limits of detection and quantification were 2.8 and 8.5 ng mL^-1, respectively. High precision and accuracy indicated by relative standard deviation ≤ 5.2, and recovery values of 95.4-102.2 %. Evident of the method greenness was verified by green analytical chemistry metric tools. HPLC-FD method was successfully applied to study PGT pharmacokinetics in rats. In conclusion, this study introduces a reliable analytical method of PGT in plasma for routine use in therapeutic drug monitoring for quality assurance and clinical follow-up.

22 Apr 2025·Blood Advances

Primary cutaneous CD8+ aggressive epidermotropic cytotoxic T-cell lymphoma with novel FGFR1 fusion treated with pemigatinib

Article

Author: Gunther, Jillian ; Tefft, Kristy ; Tang, Guilin ; Iyer, Swaminathan ; Curry, Jonathan L. ; Choi, Jaehyuk ; Miranda, Roberto N. ; Sumit, Gaur ; Torres-Cabala, Carlos A. ; Dabaja, Bouthaina ; Zhang, Yue ; Yescas, Julia Arreola ; Nardi, Valentina ; Huen, Auris

01 Apr 2025·MOLECULAR DIVERSITY

Targeting AFP-RARβ complex formation: a potential strategy for treating AFP-positive hepatocellular carcinoma

Article

Author: Dev, Radul R ; Soman, Sowmya ; Banjan, Bhavya ; Raju, Rajesh ; Vishwakarma, Riya ; Rehman, Niyas ; Kalath, Haritha ; Kumar, Pankaj ; Revikumar, Amjesh ; Abhinand, Chandran S ; Ramakrishnan, Krishnapriya

Alpha-fetoprotein (AFP) is a glycoprotein primarily expressed during embryogenesis, with declining levels postnatally. Elevated AFP levels correlate with pathological conditions such as liver fibrosis, cirrhosis, and hepatocellular carcinoma (HCC). Recent investigations underscore AFP's intracellular role in HCC progression, wherein it forms complexes with proteins like Phosphatase and tensin homolog (PTEN), Caspase 3 (CASP3), and Retinoic acid receptors and Retinoid X receptors (RAR/RXR). RAR and RXR regulate gene expression linked to cell death and tumorigenesis in normal physiology. AFP impedes RAR/RXR dimerization, nuclear translocation, and function, promoting gene expression favoring cancer progression in HCC that provoked us to target AFP as a drug candidate. Despite extensive studies, inhibitors targeting AFP to disrupt complex formation and activities remain scarce. In this study, employing protein-protein docking, amino acid residues involved in AFP-RARβ interaction were identified, guiding the definition of AFP's active site for potential inhibitor screening. Currently, kinase inhibitors play a significant role in cancer treatment and, the present study explores the potential of repurposing FDA-approved protein kinase inhibitors to target AFP. Molecular docking with kinase inhibitors revealed Lapatinib as a candidate drug of the AFP-RARβ complex. Molecular dynamics simulations and binding energy calculations, employing Mechanic/Poisson-Boltzmann Surface Area (MM-PBSA), confirmed Lapatinib's stability with AFP. The study suggests Lapatinib's potential in disrupting the AFP-RARβ complex, providing a promising avenue for treating molecularly stratified AFP-positive HCC or its early stages.

166

News (Medical) associated with Pemigatinib

08 Apr 2025

·www.prnewswire.com

TIBSOVO's Continued Success Solidifies Its Position as a Leader in IDH1 Mutant Space | DelveInsight

With its ability to specifically inhibit the mutant IDH1 enzyme, TIBSOVO addresses an unmet need in personalized cancer treatment, offering new hope for patients with limited options. As clinical evidence continues to support its efficacy and safety, TIBSOVO is positioned for strong growth in the oncology market, especially with expanding indications and global market penetration. LAS VEGAS, April 8, 2025 /PRNewswire/ -- DelveInsight's " TIBSOVO Market Size, Forecast, and Market Insight Report" highlights the details around TIBSOVO, the first and only targeted therapy approved for patients with previously treated IDH1-mutated cholangiocarcinoma. The report provides product descriptions, patent details, and competitor products (marketed and emerging therapies) of TIBSOVO. The report also highlights the historical and forecasted sales from 2020 to 2034 segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan]. Servier's TIBSOVO (ivosidenib) Overview TIBSOVO (ivosidenib) is a small molecule developed by Servier, acts as an isocitrate dehydrogenase-1 (IDH1) inhibitor. It is indicated for patients with a susceptible IDH1 mutation, which is identified through an FDA-approved test. Currently, TIBSOVO is approved for the treatment of newly diagnosed acute myeloid leukemia (AML), relapsed or refractory AML, relapsed or refractory myelodysplastic syndromes (MDS), and locally advanced or metastatic cholangiocarcinoma. According to Servier's pipeline, TIBSOVO is also being actively investigated for a range of additional indications, with key areas of exploration including solid tumors and hematological malignancies. Learn more about TIBSOVO projected market size for AML, cholangiocarcinoma, and MDS @ TIBSOVO Market Potential The oncology market has experienced significant growth in recent years, driven by advances in cancer research, the development of targeted therapies, and the increasing global prevalence of cancer. As the second-leading cause of death worldwide, cancer continues to pose a major public health challenge, creating a substantial demand for innovative treatments. The market is marked by a shift toward personalized medicine, where therapies are tailored to an individual's genetic profile, allowing for more effective and less toxic treatments. Immunotherapies, such as checkpoint inhibitors, CAR-T cell therapies, and oncolytic virus therapies, are at the forefront of these advancements, offering new hope to patients with previously untreatable cancers. Moreover, the oncology market is expanding due to the growing adoption of precision oncology and molecular diagnostics, which enable earlier and more accurate detection of cancer. This has led to increased investments in research and development, especially in areas like liquid biopsies, companion diagnostics, and next-generation sequencing. The demand for more effective and accessible cancer treatments is fueling innovation in both the pharmaceutical and biotechnology sectors, with numerous new drugs and therapies entering clinical trials and moving toward commercialization. As a result, the oncology market is poised to continue growing, with significant opportunities for breakthrough therapies in immuno-oncology, targeted treatments, and gene therapies. DelveInsight has expertise in the oncology market with an experienced team handling the oncology domain proficiently. DelveInsight has released a series of epidemiology-based market reports on Acute Myeloid Leukemia, Cholangiocarcinoma, and Myelodysplastic Syndrome. These reports include a comprehensive understanding of current treatment practices, emerging drugs, market share of individual therapies, and current and forecasted market size from 2020 to 2034, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan]. Gain deeper insights into these diseases with a customized report tailored to your needs. Connect with us today—our experts are ready to assist you! Emerging Competitors of TIBSOVO TIBSOVO faces significant competition in the newly diagnosed AML market from several alternative treatments. Oral therapies like VENCLEXTA (venetoclax), which inhibits BCL-2, and DAURISMO (glasdegib), which targets the hedgehog pathway, present strong contenders. Additionally, emerging therapies such as Lisaftoclax (Ascentage Pharma), another BCL-2 inhibitor, and gilteritinib (Astellas), a kinase inhibitor, are expected to further challenge TIBSOVO in the newly diagnosed AML space. In the R/R AML market, TIBSOVO competes with several therapies, including XOSPATA (gilteritinib), IDHIFA (enasidenib), and others. Emerging therapies such as Ziftomenib (Kura Oncology) and RVU-120 (Ryvu Therapeutics) are also expected to compete with TIBSOVO upon their approval. Ziftomenib, a small molecule developed by Kura Oncology, is currently in Phase I/II trials. In the relapsed/refractory (R/R) MDS market, TIBSOVO faces competition from therapies such as RYTELO (Imetelstat), an IV injection that inhibits oligonucleotide telomerase, and REBLOZYL (Luspatercept), a subcutaneous injection, among other treatment options. In the metastatic cholangiocarcinoma market, TIBSOVO faces relatively less competition, as there are few approved therapies. One such competitor is PEMAZYRE (pemigatinib), a kinase inhibitor that was approved in the US in 2020 and in Europe in 2021. Whereas, Tyra Biosciences' TYRA-200, an FGFR1/2/3 inhibitor, is in phase I stage of development that might be a threat to TIBSOVO one approved. To know more about the number of competing drugs in development, visit @ TIBSOVO Market Positioning Compared to Other Drugs Key Developmental Activities of TIBSOVO In October 2023, Servier announced that the FDA has approved TIBSOVO (ivosidenib tablets) for the treatment of IDH1-mutated R/R MDS. In May 2023, Servier announced that the European Commission has approved TIBSOVO as a targeted therapy in two indications: in combination with azacitidine for the treatment of adult patients with newly diagnosed AML with an isocitrate dehydrogenase-1 R132 mutation who are not eligible to receive standard induction chemotherapy; as well as in monotherapy for the treatment of adult patients with locally advanced or metastatic cholangiocarcinoma with an IDH1 R132 mutation who were previously treated by at least one prior line of systemic therapy. In May 2022, Servier announced FDA approval of TIBSOVO (ivosidenib tablets) in combination with azacitidine for patients with newly diagnosed IDH1-mutated acute myeloid leukemia. In August 2021, Servier announced FDA approval for TIBSOVO (ivosidenib tablets) for the treatment of adult patients with previously treated, locally advanced, or metastatic cholangiocarcinoma with an IDH1 mutation as detected by an FDA-approved test. TIBSOVO is the first and only targeted therapy approved for patients with previously treated IDH1-mutated cholangiocarcinoma. In May 2019, Agios announced FDA approval of TIBSOVO (ivosidenib tablets) as monotherapy for newly diagnosed adult patients with IDH1 mutant acute myeloid leukemia not eligible for intensive chemotherapy. In July 2018, the FDA approved TIBSOVO (ivosidenib) for relapsed or refractory acute myeloid leukemia with an idh1 mutation. In February 2018, the FDA accepted a new drug application and granted priority review for ivosidenib in relapsed or refractory AML with an IDH1 mutation. In December 2017, Agios submitted an NDA to the FDA for ivosidenib for the treatment of patients with relapsed/refractory aml and an IDH1 mutation. TIBSOVO Patent Details TIBSOVO is a drug owned by Servier Pharmaceuticals. Currently, it is protected by 10 Orange book-listed patents filed from 2018 to 2023, out of which none have expired yet. It is also protected by ODE exclusivity for various indications. Discover how TIBSOVO is shaping the blood cancer treatment landscape @ TIBSOVO Side Effects TIBSOVO Market Dynamics In recent years, the therapeutic landscape has undergone significant changes, with promising approaches including immunotherapy, chemotherapy combined with targeted treatments, and the regulation of immune checkpoint-mediated signaling pathways. One such treatment, Ivosidenib, works by inducing the bone marrow to develop normal blood cells, helping to reduce the frequency of blood transfusions. Investment in the research and development of drugs has surged, contributing to a rich pipeline and forecasting promising opportunities in the treatment markets for AML and cholangiocarcinomas. Therapies like TIBSOVO have the potential to capture market attention due to their efficacy and innovation. As healthcare spending continues to rise globally, the pharmaceutical market is expected to expand, providing greater opportunities for market penetration by manufacturers and facilitating the development and distribution of new treatments. Despite progress in cancer treatment, significant gaps remain in understanding the full process that governs carcinogenesis and resistance to treatments, particularly in cancers like acute myeloid leukemia (AML). This highlights the need for more advanced research to address these challenges. Additionally, the limited biomarker testing for patients with the IDH1 mutation restricts the targeted patient pool, posing a challenge for therapies like TIBSOVO to achieve their expected market share. The increasing competition from emerging therapies, such as KEYTRUDA (pembrolizumab), IMFINZI (durvalumab), and the combination of Azacitidine + Venetoclax, further intensifies the pressure on TIBSOVO. Moreover, the difficulty in obtaining sufficient patient pools and diagnostic samples during disease recurrence presents significant hurdles to the effective development of translational research, potentially slowing progress in optimizing treatments. Dive deeper to get more insight into TIBSOVO's strengths & weaknesses relative to competitors @ TIBSOVO Market Drug Report Table of Contents Related Reports Acute Myeloid Leukemia Market Acute Myeloid Leukemia Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key AML companies including Agios Pharmaceuticals, Abbvie, Jazz Pharmaceuticals, Astellas Pharma, Pfizer, Novartis Oncology, Daiichi Sankyo, AstraZeneca, Astex Pharmaceuticals, Inc., Chimerix, Takeda, Rafael Pharmaceuticals Inc., Delta-Fly Pharma, GlycoMimetics Incorporated, BerGenBio ASA, MacroGenics, Syndax Pharmaceuticals, Arog Pharmaceuticals, Forma Therapeutics, Sellas Life Sciences Group, Actinium Pharmaceuticals, Clear Creek Bio Inc., CellCentric Ltd., Biosight Ltd., Astex Pharmaceuticals, Inc., Curis, Inc., NexImmune Inc., Immunomedics, Inc., Sumitomo Dainippon Pharma Co., Ltd., Incyte Corporation, Aprea Therapeutics, Immunicum, DCPrime BV, GT Biopharma, Inc., Hanmi Pharmaceutical Company Limited, Cardiff Oncology, Bio-Path Holdings, Actinium Pharmaceuticals, Aptevo Therapeutics, Synimmune GmbH, GEMoaB Monoclonals, ImmunoGen, Inc., AGC Biologics S.p.A., Precigen, Inc., Novartis Pharmaceuticals, Xencor, Inc., Novartis, Celgene, Bristol Myers Squibb, among others. Relapsed/Refractory Acute Myeloid Leukemia Market Relapsed/Refractory Acute Myeloid Leukemia Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key R/R AML companies including Agios Pharmaceuticals, Abbvie, Jazz Pharmaceuticals, Astellas Pharma, Pfizer, Novartis Oncology, Daiichi Sankyo, AstraZeneca, Astex Pharmaceuticals, Inc., Chimerix, Takeda, Rafael Pharmaceuticals Inc., Delta-Fly Pharma, GlycoMimetics Incorporated, BerGenBio ASA, MacroGenics, Syndax Pharmaceuticals, Arog Pharmaceuticals, Forma Therapeutics, Sellas Life Sciences Group, Actinium Pharmaceuticals, Clear Creek Bio Inc., CellCentric Ltd., Biosight Ltd., Astex Pharmaceuticals, Inc., Curis, Inc., NexImmune Inc., Immunomedics, Inc., Sumitomo Dainippon Pharma Co., Ltd., Incyte Corporation, Aprea Therapeutics, Immunicum, DCPrime BV, GT Biopharma, Inc., Hanmi Pharmaceutical Company Limited, Cardiff Oncology, Bio-Path Holdings, Actinium Pharmaceuticals, Aptevo Therapeutics, Synimmune GmbH, GEMoaB Monoclonals, ImmunoGen, Inc., AGC Biologics S.p.A., Precigen, Inc., Novartis Pharmaceuticals, Xencor, Inc., Novartis, Celgene, Bristol Myers Squibb, among others. Myelodysplastic Syndrome Market Myelodysplastic Syndrome Market Insight, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of market trends, market drivers, market barriers, and key MDS companies such as Bristol-Myers Squibb, Astex Pharmaceutical, Taiho Oncology, Fibrogen, AbbVie, Gilead Sciences, Novartis, Syros Pharmaceuticals, Takeda, Pfizer, Geron Corporation, Karyopharm Therapeutics, Antengene Corporation, BerGenBio ASA, Jazz Pharmaceuticals, Aprea Therapeutics, Sanofi, Medac, among others. Cholangiocarcinoma Market Cholangiocarcinoma Market Insight, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of market trends, market drivers, market barriers, and key cholangiocarcinoma companies such as AstraZeneca, Decalth Systems, Basilea Pharmaceutica, Taiho Oncology, Eisai Pharmaceuticals, TransThera Sciences, Incyte Corporation, Roche, Agios Pharmaceuticals, Servier Pharma, 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 . 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

Drug ApprovalImmunotherapyPhase 1Priority Review

28 Mar 2025

·www.fiercepharma.com

Eli Lilly's Alzheimer's contender Kisunla gets thumbs-down from EU regulators over safety concerns

At its latest meeting, the European Medicines Agency’s Committee for Medicinal Products for Human Use backed five new medicines. After failing to pass muster with England’s drug reimbursement watchdog earlier this month, Eli Lilly’s Alzheimer’s disease med Kisunla (donanemab) also got a rejection from EU regulators.European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) recommended against a marketing authorization for the drug’s use in early symptomatic Alzheimer’s disease. The agency argued that the benefits of the treatment “were not large enough” to outweigh the risk of potentially fatal events such as swelling and bleeding of the and brain, a side effect known as amyloid-related imaging abnormalities (ARIA).Lilly will seek a re-examination of the decision. The company hopes to “continue our discussions with the agency to bring donanemab to the millions of people across Europe suffering from this relentless, fatal disease,” EVP and president of Lilly International Ilya Yuffa said in a company statement Friday. The drugmaker “remains confident in the safety and effectiveness of donanemab,” As Eisai and Biogen can attest, European regulators are cautious about the safety concerns of Alzheimer’s drugs. The partners’ Kisunla-rivaling Leqembi eventually won CHMP’s favor in February, albeit in a restricted patient population of those with mild Alzheimer’s-related cognitive impairment or dementia who carry one or no copies of the ApoE4 gene.The positive opinion there came after the regulator first denied the treatment, citing benefits that “do not counteract the risk of serious adverse events” such as ARIA. The ARIA safety risk is greater in those who have two copies of the ApoE4 gene. Over in England, NICE recently declined to recommend both of the Alzheimer’s drugs for coverage in the region’s National Health Service for the second time, despite both winning approval from the U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA). The agency cited a review from an independent committee on additional clinical data that ultimately confirmed that the medicines “are not currently cost effective.”In the U.S., both Kisunla and Leqembi are FDA-approved with a black boxed warning label for ARIA risk. Meanwhile with CHMP, several other drugmakers such as Bristol Myers Squibb received good news. The committee issued a positive opinion on the company’s subcutaneous formulation of blockbuster oncology drug Opdivo, clearing the way for an EU approval. Seven drugs—including AstraZeneca’s Calequence, Johnson & Johnson’s Tremfya and Seqirus’ flu vaccine Flucelvax—won label extensions, while Incyte’s Pemazyre was not recommended for one.The committee also wrapped up its review of Orexigen Therapeutics’ Currax-partnered weight loss med Mysimba, which was being evaluated again due to concerns of potential long-term cardiovascular risks. While CHMP determined that the benefits of the drug outweigh the risks, the company must provide more information on long-term use through an ongoing study that should wrap up in 2028. Since the available data only shows that there are no safety concerns when Mysimba is used for up to 12 months, the committee warned that treatment should be stopped after one year if weight loss of at least 5% of initial body weight isn’t maintained, and that doctors should closely monitor a patient’s yearly progress on the med. The drug was first approved in Europe in 2015.

Drug ApprovalVaccine

28 Mar 2025

·endpts.com

CHMP recommends seven label expansions and one new approval, rejects Lilly Alzheimer's drug

Bristol Myers Squibb, BeiGene, AstraZeneca and Pfizer secured European backing for label expansions, and a French pharma got its first drug approval recommendation, in a list of actions by the European Medicines Agency’s human medicines committee (CHMP) on Friday. The CHMP recommended that the European Commission expand the label for Bristol Myers Squibb’s PD-1 checkpoint inhibitor Opdivo, and also recommended the approval of the subcutaneous form of the cancer drug. The move comes a few months after the FDA approved the subcutaneous version . CHMP also recommended a new 600 mg dosage. Opdivo is currently approved as an IV infusion in Europe and has racked up at least 12 approvals in different cancers. The committee also recommended that BeiGene’s Tevimbra be expanded to use as a first-line treatment of adult patients with extensive-stage small cell lung cancer. BeiGene recently won FDA approval for Tevimbra for use in combination with platinum-based chemotherapy in adults with first-line unresectable or metastatic esophageal squamous cell carcinoma. This came after facing multiple delays due to FDA inspection pushbacks. Other CHMP label expansion recommendations include: The committee also made recommendations on several new drugs, including Averoa’s medicine Xoanacyl as a treatment for iron deficiency and hyperphosphatemia in patients with chronic kidney disease. Xoanacyl would be the company’s first marketed drug. The CHMP also recommended approving: The committee recommended rejecting Lilly’s Alzheimer’s drug Kisunla , following a pattern of skepticism of new Alzheimer’s treatments. CHMP also suggested not extending the label for Incyte’s drug Pemazyre for myeloid/lymphoid neoplasms with fibroblast growth factor receptor 1 mutations. The agency rejected the label extension over lack of “sufficiently comprehensive” data, stating the company did not compare Pemazyre with another drug or a placebo, and said the study had a limited number of patients. Pemazyre was approved by the agency in 2021 as a treatment for bile duct cancer.

Drug Approval

100 Deals associated with Pemigatinib

External Link

KEGG	Wiki	ATC	Drug Bank
D11417	Pemigatinib	L01XE	DB15102

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Advanced Bile Duct Carcinoma	South Korea	Handok, Inc.	25 Apr 2023
Chromosome 8p11 myeloproliferative syndrome	United States	Incyte Corp.	26 Aug 2022
Bile Duct Neoplasms	Canada	Incyte Corp.	17 Sep 2021
Locally Advanced Cholangiocarcinoma	European Union	Incyte Biosciences Distribution BV	26 Mar 2021
Locally Advanced Cholangiocarcinoma	Iceland	Incyte Biosciences Distribution BV	26 Mar 2021
Locally Advanced Cholangiocarcinoma	Liechtenstein	Incyte Biosciences Distribution BV	26 Mar 2021
Locally Advanced Cholangiocarcinoma	Norway	Incyte Biosciences Distribution BV	26 Mar 2021
Metastatic Cholangiocarcinoma	European Union	Incyte Biosciences Distribution BV	26 Mar 2021
Metastatic Cholangiocarcinoma	Iceland	Incyte Biosciences Distribution BV	26 Mar 2021
Metastatic Cholangiocarcinoma	Liechtenstein	Incyte Biosciences Distribution BV	26 Mar 2021
Metastatic Cholangiocarcinoma	Norway	Incyte Biosciences Distribution BV	26 Mar 2021
FGFR2 fusion or rearranged Cholangiocarcinoma	Japan	Incyte Corp.	23 Mar 2021
FGFR2 positive Intrahepatic Cholangiocarcinoma	United States	Incyte Corp.	17 Apr 2020

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Unresectable Intrahepatic Cholangiocarcinoma	Phase 3	United States	Incyte Corp.	03 Jun 2019
Unresectable Intrahepatic Cholangiocarcinoma	Phase 3	China	Incyte Corp.	03 Jun 2019
Unresectable Intrahepatic Cholangiocarcinoma	Phase 3	Japan	Incyte Corp.	03 Jun 2019
Unresectable Intrahepatic Cholangiocarcinoma	Phase 3	Austria	Incyte Corp.	03 Jun 2019
Unresectable Intrahepatic Cholangiocarcinoma	Phase 3	Belgium	Incyte Corp.	03 Jun 2019
Unresectable Intrahepatic Cholangiocarcinoma	Phase 3	Canada	Incyte Corp.	03 Jun 2019
Unresectable Intrahepatic Cholangiocarcinoma	Phase 3	Denmark	Incyte Corp.	03 Jun 2019
Unresectable Intrahepatic Cholangiocarcinoma	Phase 3	Finland	Incyte Corp.	03 Jun 2019
Unresectable Intrahepatic Cholangiocarcinoma	Phase 3	France	Incyte Corp.	03 Jun 2019
Unresectable Intrahepatic Cholangiocarcinoma	Phase 3	Germany	Incyte Corp.	03 Jun 2019

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04949191 (CTgov)	Phase 2	Neoplasms	10	Pemigatinib (Pemigatinib 6 mg Monotherapy)	coowihmpbx = lmofduqoso toxpecwqhl (ckzpfsgozt, sabtljhldy - igvmosecgx) View more	-	19 Sep 2024
				Pemigatinib (Pemigatinib 9 mg Monotherapy)	coowihmpbx = wlkzthjztm toxpecwqhl (ckzpfsgozt, jdotykpchh - okvqokdskx) View more
NCT05253807 (FIGHT-210, CTgov)	Phase 2	Recurrent Lung Small Cell Carcinoma \| Advanced Lung Non-Small Cell Carcinoma	8	Pemigatinib	nkwbjblrie = ghzlbzzrdv pmtoulxhnj (rflskyrbyv, yszidsmqoy - ihjlutgsma) View more	-	06 Aug 2024
NCT03011372 (FIGHT-203, EHA2024) Manual	Phase 2	Chromosome 8p11 myeloproliferative syndrome FGFR1 gene rearrangement	47	Pemigatinib 13.5 mg once daily (intermittent dose [ID; 2 weeks on/1 week off]	fncladqrqh(btjerpgnlx) = kqibmhvgpz aosamdmqnw (coerxetmhx ) View more	Positive	14 May 2024
NCT03822117 (FIGHT-207, Pubmed) Manual	Phase 2	Solid tumor Second line FGFR1-FGFR3 alterations	107	Pemigatinib	amyhyfsdht(quvetfpgwk) = wzrnjqddid girmdzafaj (ttyiohjuni ) View more	Positive	06 May 2024
				pemigatinib	amyhyfsdht(quvetfpgwk) = rqjinsocuh girmdzafaj (ttyiohjuni ) View more
NCT04088188 (CTgov)	Phase 1	Unresectable Intrahepatic Cholangiocarcinoma \| Advanced biliary tract cancer \| Metastatic Cholangiocarcinoma	8	Ivosidenib+Gemcitabine+cisplatin (Arm A (Ivosidenib, Cisplatin, Gemcitabine))	tihifyiwgd = wlebagtwnz fiqwolsbnr (mfwpqsflro, rziwtzgrfy - qkpjnbpnkd) View more	-	02 May 2024
				Gemcitabine+cisplatin+Pemigatinib (Arm B (Pemigatinib, Cisplatin, Gemcitabine))	lofaomjxzb(lyqtdneqjm) = ykwmnwykax mwwrpoyioc (nsnhyleqgj, mktjhykibn - vmxcrazjen) View more
NCT04096417 (ACCRU-GI-1701, CTgov)	Phase 2	Chromosomal translocation \| Unresectable Colorectal Carcinoma \| Colorectal Cancer, Hereditary Nonpolyposis, Type 1 ... View more	14	Quality-of-Life Assessment+Pemigatinib	yttvkiylxy = fnghvlbzbn hgglvwdmob (nxciyrqvuh, svzylrnabe - sruigkdxkh) View more	-	21 Feb 2024
NCT02872714 (FIGHT-201, Pubmed) Manual	Phase 2	Transitional Cell Carcinoma FGFR3 Mutation	260	Pemigatinib 13.5 mg once daily continuously	xarzxhgnqg(zzrzctwuiy) = tkilxeofjx snirqnfbli (lfwvxufruv, 10.9 - 26.7) View more	Positive	01 Feb 2024
				Pemigatinib 13.5 mg once daily intermittently	xarzxhgnqg(zzrzctwuiy) = dwwcxuaebh snirqnfbli (lfwvxufruv, 15.5 - 32.7) View more
NCT02924376 (FIGHT-202, ASCO_GI2024) Manual	Phase 2	Bile Duct Neoplasms FGFR2 Mutation	120	Pemigatinib	sphtefrpoc(jthgvdjwrx) = mrayeputcz haypjrweyo (mnvbtmetgy, 6.4 - 8.6) View more	Positive	18 Jan 2024
NCT03822117 (FIGHT-207, AACR2023) Manual	Phase 2	Solid tumor	107	pemigatinib 13.5 mg (cohorts A)	nkptdahggz(amtxhbjqqp) = yrylmnkeun tjuqowprbg (ncfbuusmhs )	Positive	14 Apr 2023
				pemigatinib 13.5 mg (cohorts B)	nkptdahggz(amtxhbjqqp) = lyadfxwzze tjuqowprbg (ncfbuusmhs )
NCT04256980 (AACR2023) Manual	Phase 2	FGFR2 fusion or rearranged Cholangiocarcinoma Second line FGFR2	30	Pemigatinib 13.5 mg	jjxxlsclrt(oahueydnli) = pjsopptalj xuxnrtvxng (msxqylgehe, 15.2 - NC) View more	Positive	14 Apr 2023

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