To find the recommended dose of ropeginterferon alfa-2b in combination with azacitidine in patients with newly diagnosed MDS/AML with TP53 mutations. To learn if the recommended dose of ropeinterferon alfa-2b found in Dose Escalation in combination with azacitidine can help to control the disease.

Start Date27 Nov 2026

Sponsor / Collaborator

PharmaEssentia Corp.

NCT07468916

/ Not yet recruitingPhase 2IIT

Ropeginterferon Alfa-2b for MDS/MPN Overlap Syndromes, Including CMML and MDS/MPN-RS-T

This phase II trial tests the safety, best dose, and effectiveness of ropeginterferon alfa-2b for the treatment of patients with myelodysplastic syndrome/myeloproliferative neoplasm overlap syndromes and chronic myelomonocytic leukemia. Ropeginterferon alfa-2b is a form of interferon. Interferons are a type of signaling protein normally produced by the body as part of the immune response. Interferons interfere with the division of cancer cells and can slow cancer cell growth. Ropeginterferon alfa-2b is a long-acting form of a type of interferon called interferon alfa-2b. In the body, ropeginterferon alfa-2b causes the production of proteins that modulate the immune system and have anticancer effects.

Start Date29 Sep 2026

Sponsor / Collaborator

Jonsson Comprehensive Cancer Center

[+1]

NCT06290765

/ WithdrawnPhase 4

Efficacy and Safety of Ropeginterferon Alfa 2b (P1101) for Patients With Polycythemia Vera - A Randomized Open Label Global Multicenter Study (PARADIGM-PV)

This is a randomized, open-label, multicenter, two-arm study to assess the efficacy and safety of ropeginterferon alfa-2b for patients with PV. The entire study period is 60 weeks, including a main treatment phase (32 weeks), an extension treatment phase (24 weeks), and a safety follow-up phase (four weeks). However, the study may be extended for additional period of treatment after Week 60 pending the primary endpoint analysis at Week 32. Approximately 70 patients with PV will be enrolled.

Start Date01 Aug 2026

Sponsor / Collaborator

PharmaEssentia Corp.

100 Clinical Results associated with Ropeginterferon alfa-2b-NJFT

100 Translational Medicine associated with Ropeginterferon alfa-2b-NJFT

100 Patents (Medical) associated with Ropeginterferon alfa-2b-NJFT

253

Literatures (Medical) associated with Ropeginterferon alfa-2b-NJFT

01 Sep 2026·Lancet regional health. Americas

Ropeginterferon alfa-2b-njft treatment in essential thrombocythemia across different driver mutations: results from a North American, single-arm, multicentre study (EXCEED-ET)

Article

Author: Fang, Bruno ; Tashi, Tsewang ; Oh, Stephen T ; Abu-Zeinah, Ghaith ; Goel, Swati ; Vachhani, Pankit ; Masarova, Lucia ; Safah, Hana ; Mascarenhas, John ; Mesa, Ruben A ; How, Joan ; O'Connell, Casey L ; Zagrijtschuk, Oleh ; El Chaer, Firas ; Cunningham, Julia M ; Cerquozzi, Sonia ; Babu, Sunil ; Castro, Henry ; Halpern, Anna B ; Hunter, Anthony M ; Yacoub, Abdulraheem ; Lucas, Jennifer ; Foltz, Lynda ; Reeves, Brandi N ; Bose, Prithviraj ; Li, Zhuoyan ; Qin, Albert ; Podoltsev, Nikolai ; Rein, Lindsay ; Maze, Dawn ; Hillis, Christopher

Background:

Ropeginterferon alfa-2b-njft (ropeg), a mono-PEGylated interferon-α, showed efficacy and safety in patients with hydroxyurea-intolerant/resistant essential thrombocythemia (ET) in the phase 3 SURPASS-ET largely conducted in Asia. Here we report the results from the EXCEED-ET study, which evaluated ropeg in both treatment-naïve and hydroxyurea pre-treated ET in North America.

Methods:

The single-arm, multicentre, phase 2b trial EXCEED-ET was conducted at 28 centers in the United States and Canada. Patients with ET enrolled were required to have a platelet count >450 × 10⁹/L at screening. Patients were eligible regardless of prior cytoreductive therapy status and included both treatment-naïve and hydroxyurea-pretreated patients. Ropeg was subcutaneously administered in an accelerated titration schedule at 250 μg at Week 0, 350 μg at Week 2, and 500 μg from Week 4 and beyond. The primary endpoint was durable, modified European LeukemiaNet (ELN) response at both Months 10 and 13 (ClinicalTrials.gov identifier: NCT05482971).

Findings:

Between January 17, 2023, and May 15, 2025, 106 patients were screened and 91 were enrolled. At baseline, participant median age was 57 years (range, 22-84) with 57/91 (62.6%) being female and 71/91 (78.0%) being White. Among 91 patients enrolled, durable modified ELN response rate was 60.2% (95% CI, 49.0-71.4). The median time to hematologic response was only 8.4 weeks (95% CI: 8.1-11.9). Splenomegaly and symptom improvement or non progression occurred in 90/91 (98.9%) and 71/91 (78.0%), respectively. Seven of 91 (7.7%) patients with palpable spleen at baseline and all had an improvement in disease-related signs. The rates of molecular response for driver mutations at Month 13 as defined by ELN2009 criteria were 35.0% (7/20) for patients with JAK2V617F, 16.0% (4/25) for CALR, and 25.0% (2/8) for MPL mutations. One (1/1) TP53 mutation-positive patient showed a complete molecular response. Thrombosis and progression occurred in 3.4% (3/91) and 1.1% (1/91), respectively. Most treatment-emergent adverse events were mild, including fatigue (60%) and reversible transaminase elevations (58%). There was no grade 5 TEAE and one (1.1%) grade 4 TEAE (hepatitis toxic) which recovered without sequelae. Grade ≥3 TEAEs occurred in 27.5% (25/91) of the participants, most commonly with alanine aminotransferase and aspartate aminotransferase increases (4.4%; 4/91), neutrophil count decrease (4.4%; 4/91), fatigue (3.3%; 3/91) and lymphocyte count decrease (3.3%; 3/91). Treatment-related serious adverse events occurred in 5.5% (5/91) of patients. Pneumonia was the most common SAE, occurring in 2/91 (2.2%) patients. Ropeg-related discontinuations occurred in 9/91 (9.9%) patients.

Interpretation:

Ropeg showed efficacy and substantial molecular responses with good overall tolerability across a broad ET population.

Funding:

PharmaEssentia.

15 Jun 2026·INTERNAL MEDICINE

Effective Treatment with Ruxolitinib and Ropeginterferon Alfa-2b for Refractory TAFRO-like Syndrome

Article

Owing to the lack of appropriate treatment, TAFRO syndrome often presents with multiple organ dysfunction and fatality. The Janus kinase (JAK)/signal transducers and activators of transcription (STAT) pathway has recently been shown to play an important role in the pathogenesis of inflammation in TAFRO syndrome, and inhibitors of the JAK/STAT pathway may be effective as therapeutic agents for TAFRO syndrome. We herein report the successful treatment using combination therapy with ruxolitinib and ropeginterferon alfa-2b of a case of TAFRO-like syndrome with a long history of polycythemia vera with JAK2 V617F refractory to several treatments.

09 Jun 2026·Blood Advances

Ropeginterferon alfa-2b for prefibrotic myelofibrosis and lower-risk myelofibrosis requiring cytoreduction

Article

Author: Li, Vivian ; Wu, Tony K. Y. ; Lee, Carmen Y. Y. ; Gill, Harinder ; Yim, Rita ; Zhang, Qi ; Chin, Lynn ; Kwong, Yok-Lam ; Lee, Paul ; Au, Lester ; Leung, Garret M. K. ; Chan, Po-Yan

Abstract:

There is currently no consensus on the optimal treatment of early myelofibrosis (MF). Ropeginterferon alfa-2b (ropeg) is a next-generation monopegylated interferon alfa-2b developed specifically to treat myeloproliferative neoplasms. We conducted a phase 2 study in patients with prefibrotic primary MF (pre-PMF) and Dynamic International Prognostic Scoring System low-/intermediate-1–risk fibrotic PMF or secondary MF (SMF) who required cytoreduction. Ongoing treatment with hydroxyurea was substituted with ropeg (week 0, 250 μg; week 2, 350 μg; week 4 onward, 500 μg every 2 weeks). The primary end points were safety and clinicohematologic complete response (CHCR). Seventy-one patients (40 men and 31 women) with a median age of 60 years (range, 31-86) were enrolled and followed up for a median of 119 weeks (range, 10-131). At weeks 24, 48, and 104, CHCR was achieved in 63.8% (pre-PMF, 74%; fibrotic PMF, 20%; fibrotic SMF, 42.9%), 63.5% (pre-PMF, 69.6%; fibrotic PMF, 25%; SMF, 53.9%), and 70% of patients (pre-PMF, 78.4%; fibrotic PMF, 33.3%; SMF, 50%), respectively. Thrombo-hemorrhagic events were not observed after stopping hydroxyurea. At weeks 24, 48, and 104, reduction in JAK2V617F variant allele frequency (VAF) was observed in 53.1%, 70%, and 67.6% of patients, and reduction in CALR mutant VAF was observed in 52.6%, 42.9%, and 40% of patients, respectively. In 50 evaluable cases at week 104, 13 patients (26%) had reduced bone marrow reticulin fibrosis. There were 3 treatment discontinuations (4.2%) due to adverse events. In conclusion, ropeg was safe and induced CHCR associated with significant molecular responses in patients with early MF. This trial was registered at www.clinicaltrials.gov as NCT04988815.

News (Medical) associated with Ropeginterferon alfa-2b-NJFT

07 Aug 2026

·www.aop-health.com

Quantum Award in Favor of AOP Health in BESREMi® Case

Vienna, 07.08.2026. An ICC Arbitral Tribunal has awarded AOP Orphan Pharmaceuticals GmbH (“AOP Health”) a total of about EUR 112 Mio in a quantum award issued in the ongoing arbitration proceedings with PharmaEssentia Corp. (“PharmaEssentia”) concerning BESREMi® (ropeginterferon alfa-2b). The award quantifies AOP Health’s damage claims for PharmaEssentia’s intentional breaches at ca. EUR 82 Mio. It also awards AOP Health ca. EUR 31 Mio plus interest as reimbursement for AOP Health overpayments made to PharmaEssentia as a result of excessive pricing in the years 2019-2022. The Tribunal thereby confirmed that PharmaEssentia has been overcharging AOP Health by up to 900 % over these years. The Tribunal affirmed AOP Health's valid set-off of the profit-sharing payments amount owed to PharmaEssentia of approximately EUR 17 Mio against AOP Health's substantially exceeding damages claims. This means that AOP Health shall not make any payment to PharmaEssentia. Interest on AOP Health’s claims will continue to accrue until PharmaEssentia has made payment. All of PharmaEssentia’s remaining claims of the quantum stage of the proceedings were dismissed. The decision follows the partial final award received in February 2025, in which the ICC Arbitral Tribunal found, among other things, that PharmaEssentia had intentionally breached its contractual obligations and was liable for several claims arising out of the parties’ licensing agreement. The quantum award now determines the amounts due in relation to these claims. Tribunal confirms PharmaEssentia’s excessive pricing of up to 900% Applying the Tribunal’s reasoning in the quantum award to the product deliveries for the years 2023-2025, PharmaEssentia has overcharged AOP Health by another ca. EUR 65 Mio. After set-off with profit sharing amounts for these years, PharmaEssentia will still owe a substantial amount to AOP Health. In short, AOP Health owes no payment to PharmaEssentia. Meanwhile, PharmaEssentia’s liability towards AOP Health is currently in the three-digit EUR million range. Safeguarding Patient Access AOP Health welcomes the decision, which the company sees as a further confirmation of its position and its conduct in the proceedings. The company remains focused on ensuring stable and sustainable access to BESREMi® for patients in need. In its seventh year after its approval by European Medicines Agency (“EMA”), AOP Health has successfully made BESREMi® available to an estimated 12,600 patients in AOP Health’s licensed territory. Dr. Rudolf Widmann, one of the two founders of AOP Health, says: As with the previous award, PharmaEssentia may seek a limited review of the quantum award before the competent German courts. Proceedings concerning the review of the February 2025 merit award which was confirmed by the Higher Regional Court of Frankfurt in April 2026 are still pending before the German Federal Court of Justice. Background to the dispute The conflict concerns BESREMi®, a treatment for rare blood cancers, particularly polycythemia vera. AOP Health acquired the rights for the development and commercialization of BESREMi® in the European, Commonwealth of Independent States (CIS), and Middle Eastern markets from PharmaEssentia in 2009. Since 2017, PharmaEssentia has repeatedly attempted to terminate its agreement with AOP Health regarding BESREMi®. In October 2020, an ICC Arbitral Tribunal rejected these attempts and awarded AOP Health damages, while dismissing PharmaEssentia’s counterclaims. Subsequent set-aside proceedings confirmed the award in its essentials, while identifying procedural issues relating to the quantification of damages. In November 2020, PharmaEssentia initiated a further legal action against AOP Health, alleging damage claims. AOP Health in turn claimed damages for delays in BESREMi®’s European approval caused by PharmaEssentia and for the use of AOP Health’s clinical trial data in connection with PharmaEssentia’s U.S. marketing authorization. In February 2025, the ICC Arbitral Tribunal issued a partial final award in AOP Health’s favor on liability and dismissed PharmaEssentia’s liability claims in its entirety. The newly received quantum award now specifies the amounts due for the claims upheld in the partial final award. About BESREMi® BESREMi® is the first interferon that was approved for polycythemia vera, a myeloproliferative neoplasm (MPN), indicated in the European Union as monotherapy in adults for treatment of polycythemia vera without symptomatic enlarged spleen. Its overall safety and efficacy were demonstrated in multiple clinical studies. BESREMi® (ropeginterferon alfa-2b) is a long-acting, mono-pegylated proline interferon (ATC L03AB15). It is administered once every 2 weeks initially, or up to every 4 weeks after stabilization of blood values. BESREMi® is designed to be self-administered subcutaneously with a pre-filled pen. Please visit: European Medicines Agency Summary of Product Characteristics for: BESREMi® About AOP Health AOP Health Group is a global pharmaceutical enterprise with headquarters in Vienna. Its mission is to address high unmet medical needs through impactful, science-driven therapies and care models. Since 1996, AOP Health has pioneered research where limited therapies exist, to develop solutions for patients with rare diseases and severe conditions in niche therapeutic areas. Guided by the principle "Needs. Science. Trust.” the AOP Health Group demonstrates its strong commitment to patients and healthcare professionals through innovation and long-term partnerships, to improve patient outcomes worldwide. Press Contact. Mag. Nina Roth, MAS Head of Corporate Communications nina.roth[at]aop-health.com Photocredit Portrait: AOP Health/Daniel Ospelt

Drug ApprovalPatent InfringementLicense out/in

06 Jul 2026

·www.businesswire.com

PharmaEssentia Announces Health Canada Approval of BESREMi® (ropeginterferon alfa-2b) for Polycythemia Vera (PV)

BURLINGTON, Mass.--(BUSINESS WIRE)--PharmaEssentia Corporation (TWSE: 6446), a global biopharmaceutical innovator based in Taiwan leveraging deep expertise and proven scientific principles to deliver new biologics in hematology and oncology, today announced that its wholly owned subsidiary, FORUS Therapeutics Inc, has received a Notice of Compliance (NOC) authorizing BESREMi® (ropeginterferon alfa-2b) for the treatment of adults with polycythemia vera (PV). BESREMi® is expected to become commercially available in Canada in the coming weeks. PV is a rare, chronic blood cancer characterized by overproduction of red blood cells, which increases blood viscosity and can lead to serious complications, including thrombosis, stroke, myocardial infarction, and progression to myelofibrosis or acute myeloid leukemia. “We are very pleased that Health Canada has approved BESREMi® for the treatment of adults with PV,” said Ko-Chung Lin, Ph.D., Founder and Chief Executive Officer of PharmaEssentia. “BESREMi® is a next-generation interferon demonstrated to provide durable hematologic and molecular responses, reduced symptom burden, and a favorable safety and tolerability profile. We look forward to working closely with Canadian hematologists and other key stakeholders to make BESREMi® available to patients living with PV across Canada.” “PV is a chronic myeloproliferative neoplasm that requires long-term management to reduce the risk of complications and control symptoms. The approval of BESREMi® in Canada is an important and welcome development for patients living with PV and for the physicians who care for them,” said Shireen Sirhan, M.D., President, Canadian Myeloproliferative Neoplasms Group; Vice-President (Research), Quebec Research Group for Chronic Myeloid Leukemia and Myeloproliferative Neoplasms (GQR-LMC-NMP); Hematologist, Jewish General Hospital, McGill University Montreal. “The availability of an interferon therapy specifically approved for PV has been long anticipated within the Canadian MPN community and represents a meaningful expansion of the treatment options available to patients. This approval reflects continued progress in the field and supports a more individualized approach to the management of PV." About PharmaEssentia PharmaEssentia Corporation (TWSE: 6446), headquartered in Taipei, Taiwan, is a global and rapidly growing biopharmaceutical innovator. Leveraging deep expertise and proven scientific principles, PharmaEssentia aims to deliver effective new biologics for challenging diseases in hematology, oncology, and immunology, with one approved product and a diversifying pipeline. Founded in 2003 by a team of Taiwanese-American executives and renowned scientists from U.S. biotechnology and pharmaceutical companies, today PharmaEssentia is expanding its global presence with operations in the U.S., Japan, China, and Korea, along with a world-class biologics production facility in Taichung, Taiwan. For more information about PharmaEssentia Corp visit the website, LinkedIn or X (formerly Twitter). About Polycythemia Vera (PV) Polycythemia vera (PV) is a cancer originating from a disease-initiating stem cell in the bone marrow resulting in a chronic increase of red blood cells, white blood cells and platelets. PV may result in cardiovascular complications such as thrombosis and embolism, and often transforms to secondary myelofibrosis or leukemia. While the molecular mechanism underlying PV is still subject of intense research, current results point to a set of acquired mutations, the most important being a mutant form of JAK2. About BESREMi® (ropeginterferon alfa-2b-njft) Ropeginterferon alfa-2b-njft is currently FDA-approved and marketed as BESREMi® for the treatment of adults with polycythemia vera (PV). The Company is seeking a ropeginterferon alfa-2b-njft label expansion to include ET and has submitted an sBLA with the U.S. FDA. BESREMi® holds orphan drug designation in the United States for the treatment of polycythemia vera (PV) in adults. It has received regulatory approval in over 40 countries, including from the European Medicines Agency (2019), the U.S. Food and Drug Administration (2021), the Pharmaceuticals and Medical Devices Agency in Japan (2023), and Health Canada (2026). The product was developed by PharmaEssentia. PharmaEssentia retains full global intellectual property rights across all indications. INDICATION BESREMi® is indicated for the treatment of adults with polycythemia vera. IMPORTANT SAFETY INFORMATION WARNING: RISK OF SERIOUS DISORDERS Interferon alfa products may cause or aggravate fatal or life-threatening neuropsychiatric, autoimmune, ischemic, and infectious disorders. Patients should be monitored closely with periodic clinical and laboratory evaluations. Therapy should be withdrawn in patients with persistently severe or worsening signs or symptoms of these conditions. In many, but not all cases, these disorders resolve after stopping therapy. CONTRAINDICATIONS Existence of or history of severe depression, suicidal ideation, or suicide attempt Hypersensitivity to interferons or any inactive ingredients Moderate or severe hepatic impairment History or presence of active serious or untreated autoimmune disease History of transplantation and receiving immunosuppressant agents WARNINGS AND PRECAUTIONS Patients exhibiting the following events should be closely monitored and may require dose reduction or discontinuation of therapy: Depression and Suicide: Monitor closely for symptoms and need for treatment. Endocrine Toxicity: Discontinue if endocrine disorders occur that cannot be medically managed. Cardiovascular Toxicity: Avoid use in patients with severe, acute or unstable cardiovascular disease. Monitor patients with history of cardiovascular disorders more frequently. Decreased Peripheral Blood Counts: Perform blood counts at baseline, every 2 weeks during titration, and at least every 3-6 months during maintenance treatment. Hypersensitivity Reactions: Stop treatment and immediately manage reaction. Pancreatitis: Consider discontinuation if confirmed pancreatitis Colitis: Discontinue if signs or symptoms of colitis Pulmonary Toxicity: Discontinue if pulmonary infiltrates or pulmonary function impairment Ophthalmologic Toxicity: Advise patients to have eye examinations before and during treatment. Evaluate eye symptoms promptly and discontinue if new or worsening eye disorders. Hyperlipidemia: Monitor serum triglycerides before BESREMi® treatment and intermittently during therapy and manage when elevated. Hepatotoxicity: Monitor liver enzymes and hepatic function at baseline and during treatment. Reduce dose or discontinue depending on severity. Renal Toxicity: Monitor serum creatinine at baseline and during therapy. Discontinue if severe renal impairment develops. Dental and Periodontal Toxicity: Advise patients on good oral hygiene and to have regular dental examinations. Dermatologic Toxicity: Consider discontinuing if clinically significant dermatologic toxicity. Driving and Operating Machinery: Advise patients to avoid driving or using machinery if they experience dizziness, somnolence, or hallucination. Please see full Prescribing Information, including Boxed Warning.

Drug ApprovalOrphan Drug

26 Jun 2026

·www.biospace.com

PharmaEssentia Announces FDA Approval and U.S. Launch of BESREMi Pen™ (ropeginterferon alfa-2b-njft) for Polycythemia Vera

New pre-filled pen device enhances patient experience by simplifying self-administration BURLINGTON, Mass.--(BUSINESS WIRE)--PharmaEssentia USA Corporation, a subsidiary of PharmaEssentia Corporation (TWSE: 6446), a global biopharmaceutical innovator based in Taiwan leveraging deep expertise and proven scientific principles to deliver new biologics in hematology and oncology, today announced the U.S. Food and Drug Administration (FDA) approval and launch of the BESREMi Pen™ (ropeginterferon alfa-2b-njft) device for adult patients with polycythemia vera (PV). The device offers a more convenient self-administration option than the currently available pre-filled syringe. The BESREMi Pen™ is expected to become commercially available in the U.S. in the coming weeks. “The U.S. approval of the BESREMi Pen™ is a significant milestone both for our company, as well as the patients we serve,” said Samuel Lin, Head of Global Operations, PharmaEssentia. “With this new device, we’re empowering more people living with PV to manage their condition with greater ease and confidence. It reflects our continued commitment to delivering not only innovative therapeutics, but also smarter, more intuitive ways to support long-term care.” “I’m very excited to see the FDA approval of the BESREMi Pen™ as a new option for patients,” said Dr. John Mascarenhas, Director of the Center of Excellence for Blood Cancers and Myeloid Disorders at Mount Sinai in New York City. “Treatment consistency is critical for managing PV, and this device has the potential to make a meaningful positive impact on patients’ lives by simplifying self-administration and supporting better adherence.” About PharmaEssentia PharmaEssentia USA Corporation, located in Burlington, Massachusetts, is a subsidiary of PharmaEssentia Corporation (TWSE: 6446). PharmaEssentia Corporation, headquartered in Taipei, Taiwan, is a global and rapidly growing biopharmaceutical innovator. Leveraging deep expertise and proven scientific principles, PharmaEssentia aims to deliver effective new biologics for challenging diseases in the areas of hematology, oncology, and immunology with one approved product and a diversifying pipeline. Founded in 2003 by a team of Taiwanese-American executives and renowned scientists from U.S. biotechnology and pharmaceutical companies, today PharmaEssentia is expanding its global presence with operations in the U.S., Japan, China, and Korea, along with a world-class biologics production facility in Taichung, Taiwan. For more information about PharmaEssentia USA, visit the website , LinkedIn or X (formerly Twitter) . About Polycythemia Vera (PV) Polycythemia vera (PV) is a cancer originating from a disease-initiating stem cell in the bone marrow resulting in a chronic increase of red blood cells, white blood cells and platelets. PV may result in cardiovascular complications such as thrombosis and embolism, and often transforms to secondary myelofibrosis or leukemia. While the molecular mechanism underlying PV is still subject of intense research, current results point to a set of acquired mutations, the most important being a mutant form of JAK2. About BESREMi® (ropeginterferon alfa-2b-njft) Ropeginterferon alfa-2b-njft is currently FDA-approved and marketed as BESREMi® for the treatment of adults with polycythemia vera (PV). The Company plans to seek a ropeginterferon alfa-2b-njft label expansion to include ET and has submitted a sBLA with the U.S. FDA. BESREMi® holds orphan drug designation in the United States for the treatment of polycythemia vera (PV) in adults. It has received regulatory approval in over 40 countries, including from the European Medicines Agency (2019), the U.S. Food and Drug Administration (2021), and the Pharmaceuticals and Medical Devices Agency in Japan (2023). The product was developed by PharmaEssentia. PharmaEssentia retains full global intellectual property rights across all indications. INDICATION BESREMi® is indicated for the treatment of adults with polycythemia vera. IMPORTANT SAFETY INFORMATION WARNING: RISK OF SERIOUS DISORDERS Interferon alfa products may cause or aggravate fatal or life-threatening neuropsychiatric, autoimmune, ischemic, and infectious disorders. Patients should be monitored closely with periodic clinical and laboratory evaluations. Therapy should be withdrawn in patients with persistently severe or worsening signs or symptoms of these conditions. In many, but not all cases, these disorders resolve after stopping therapy. CONTRAINDICATIONS Existence of or history of severe depression, suicidal ideation, or suicide attempt Hypersensitivity to interferons or any inactive ingredients Moderate or severe hepatic impairment History or presence of active serious or untreated autoimmune disease History of transplantation and receiving immunosuppressant agents WARNINGS AND PRECAUTIONS Patients exhibiting the following events should be closely monitored and may require dose reduction or discontinuation of therapy: Depression and Suicide: Monitor closely for symptoms and need for treatment. Endocrine Toxicity: Discontinue if endocrine disorders occur that cannot be medically managed. Cardiovascular Toxicity: Avoid use in patients with severe, acute or unstable cardiovascular disease. Monitor patients with history of cardiovascular disorders more frequently. Decreased Peripheral Blood Counts: Perform blood counts at baseline, every 2 weeks during titration, and at least every 3-6 months during maintenance treatment. Hypersensitivity Reactions: Stop treatment and immediately manage reaction. Pancreatitis: Consider discontinuation if confirmed pancreatitis Colitis: Discontinue if signs or symptoms of colitis Pulmonary Toxicity: Discontinue if pulmonary infiltrates or pulmonary function impairment Ophthalmologic Toxicity: Advise patients to have eye examinations before and during treatment. Evaluate eye symptoms promptly and discontinue if new or worsening eye disorders. Hyperlipidemia: Monitor serum triglycerides before BESREMi® treatment and intermittently during therapy and manage when elevated. Hepatotoxicity: Monitor liver enzymes and hepatic function at baseline and during treatment. Reduce dose or discontinue depending on severity. Renal Toxicity: Monitor serum creatinine at baseline and during therapy. Discontinue if severe renal impairment develops. Dental and Periodontal Toxicity: Advise patients on good oral hygiene and to have regular dental examinations. Dermatologic Toxicity: Consider discontinuing if clinically significant dermatologic toxicity. Driving and Operating Machinery: Advise patients to avoid driving or using machinery if they experience dizziness, somnolence, or hallucination. Please see full Prescribing Information , including Boxed Warning. Contacts Media Muriel Huang Director, Investor Relations and Corporate Communication muriel_huang@pharmaessentia.com

Drug ApprovalOrphan Drug

100 Deals associated with Ropeginterferon alfa-2b-NJFT

External Link

KEGG	Wiki	ATC	Drug Bank
D11027	Ropeginterferon alfa-2b-NJFT	L03AB15	DB15119

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Polycythemia Vera	European Union	AOP Orphan Pharmaceuticals GmbH	15 Feb 2019
Polycythemia Vera	Iceland	AOP Orphan Pharmaceuticals GmbH	15 Feb 2019
Polycythemia Vera	Liechtenstein	AOP Orphan Pharmaceuticals GmbH	15 Feb 2019
Polycythemia Vera	Norway	AOP Orphan Pharmaceuticals GmbH	15 Feb 2019

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Primary Myelofibrosis	Phase 3	United States	PharmaEssentia Corp.	18 Jul 2025
Primary Myelofibrosis	Phase 3	China	PharmaEssentia Corp.	18 Jul 2025
Primary Myelofibrosis	Phase 3	Japan	PharmaEssentia Corp.	18 Jul 2025
Primary Myelofibrosis	Phase 3	Canada	PharmaEssentia Corp.	18 Jul 2025
Primary Myelofibrosis	Phase 3	Hong Kong	PharmaEssentia Corp.	18 Jul 2025
Primary Myelofibrosis	Phase 3	Jordan	PharmaEssentia Corp.	18 Jul 2025
Primary Myelofibrosis	Phase 3	Singapore	PharmaEssentia Corp.	18 Jul 2025
Primary Myelofibrosis	Phase 3	South Korea	PharmaEssentia Corp.	18 Jul 2025
Primary Myelofibrosis	Phase 3	Taiwan Province	PharmaEssentia Corp.	18 Jul 2025
Thrombocythemia, Essential	Phase 3	United States	PharmaEssentia Corp.	25 Aug 2020

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT05482971 (EXCEED-ET, Pubmed \| Lancet Reg Health Am)	Phase 2	Thrombocythemia, Essential	91	Ropeginterferon alfa-2b-njft	twtylfysne(dnsvcsusfd) = ridvgckbmg twzmbsxfip (fknimfgzwh, 49.0 - 71.4) View more	Positive	01 Sep 2026
ASCO2026	Not Applicable	Polycythemia Vera	18	Ropeginterferon alfa-2b with HIDAT	yckgjpkirk(cvizzixdbm) = podzlwfxll nyoxknkclx (vajrksvpbq ) View more	Positive	29 May 2026
				Ropeginterferon alfa-2b with low-dose regimens	yckgjpkirk(cvizzixdbm) = jeghmewfkv nyoxknkclx (vajrksvpbq ) View more
EHA2026	Not Applicable	Polycythemia Vera JAK2V617F	15	Ropeginterferone alfa-2b (JAK2V617F positive + Polycythemia Vera + HU-intolerant)	fizlqvrfet(emzeqeyiph) = mxsibncsxc powcfwnpve (onunhijsoa ) View more	Positive	12 May 2026
NCT06514807 (ROP-ET, EHA2026)	Phase 3	Thrombocythemia, Essential	132	Ropeginterferon alfa-2b	ngtpwsxrut(yqauamfecf) = clmparfgtn xspzmkhpbb (xqnyhhgtfh ) View more	Positive	12 May 2026
				Ropeginterferon alfa-2b (high‐risk)	ucwcqojzqj(cnnuymxdke) = apmqusomsp ltzjakavea (wyudystlcm, 42.3 - 69.7)
NCT06506084 (EHA2026)	Not Applicable	Polycythemia Vera	262	ROPEGINTERFERON ALFA-2B (HU-intolerant patients)	tqvemwkcdx(nwtuqfqvuv) = ktskntoixa gfpvgiqrtb (naoytwqzmp ) View more	Positive	12 May 2026
				ROPEGINTERFERON ALFA-2B (with a history of skin cancer)	tqvemwkcdx(nwtuqfqvuv) = xyxvjlswdl gfpvgiqrtb (naoytwqzmp ) View more
SURPASS-ET (EHA2026)	Phase 3	Thrombocythemia, Essential	137	Ropeginterferon alfa-2b (SURPASS-ET study)	bfnobcbyti(adnwpgtfac) = hyzxfajnxg dmmceibrky (hqocfhzpsz ) View more	Positive	12 May 2026
				Anagrelide (SURPASS-ET study)	zcmrwjinbq(ujboxpjamf) = uakhzeoerp ldquzbuyie (niyxifrqyy ) View more
EHA2026	Not Applicable	Polycythemia Vera	99	Ropeginterferon alfa-2b	qodmftwqkb(vffjhmesgx) = elvoozksqs kjzhjqqpjv (qicwevacgf ) View more	Positive	12 May 2026
NCT04285086 (SURPASS-ET, EHA2026)	Phase 3	Thrombocythemia, Essential	174	P1101	yopjsngsdx(nuujadjadv) = suotkqamyp yrilzhsttx (uywvkyujrk, 66.4 - 84.4) View more	Positive	12 May 2026
				Anagrelide	yopjsngsdx(nuujadjadv) = xwswjjkfle yrilzhsttx (uywvkyujrk, 30.6 - 55.0) View more
EHA2026	Not Applicable	Myeloproliferative Disorders	206	ropeginterferon alfa-2b	ufixpoasnx(zbwhrqbspx) = Adverse event-related discontinuations included hepatic dysfunction, neuropathy, migraine, ocular symptoms, and vascular events, each occurring in ≤ 2 patients. btbqpsywwy (duoucymvgw ) View more	Positive	12 May 2026
				ropeginterferon alfa-2b (switch cohort)
EBMT2026	Not Applicable	Polycythemia Vera	369	Ropeginterferon alfa-2b	bgiaqbfuaw(cikgrtnchj) = pooled incidences of thrombosis (4.0%), serious adverse events (5.0%), treatment discontinuation (7.0%), and disease transformation (1.0%), with no consistent signal of increased toxicity associated with rapid dose escalation. dxjsfhepro (nxavjbifcr )	Positive	22 Mar 2026
				Ropeginterferon alfa-2b (slow dose-up)

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