Ropeginterferon alfa-2b-NJFT

First global approval of BESREMi ® in essential thrombocythemia supports continued expansion of the PharmaEssentia MPN franchise; U.S. PDUFA date remains on track for August 30, 2026 BESREMi ® becomes first new approved therapy for ET in nearly 30 years 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 that the Taiwan's Ministry of Health and Welfare (MOHW) has approved ropeginterferon alfa-2b-njft (BESREMi ® ) for the treatment of adult patients with essential thrombocythemia (ET). BESREMi ® is the first new therapy approved for ET in nearly 30 years. The approval represents the first global regulatory approval of BESREMi ® in ET and marks an important milestone in the strategy to expand BESREMi across myeloproliferative neoplasms (MPNs). With the first global approval in ET now secured in Taiwan, BESREMi ® is well-positioned to address a significant global market opportunity, including in the United States, where the U.S. Food and Drug Administration (FDA) is currently reviewing a supplemental Biologics License Application (sBLA) for ET with a Prescription Drug User Fee Act (PDUFA) target action date of August 30, 2026. ET is a chronic MPN characterized by excessive platelet production and increased risk of thrombosis, hemorrhage. Based on data from the Phase 3 SURPASS-ET and Phase 2 EXCEED-ET studies, PharmaEssentia believes BESREMi ® has the potential to address a broad ET patient population regardless of underlying disease subtype, supporting a significant global expansion opportunity for the BESREMi ® franchise. “This first global approval of BESREMi ® in ET represents an important strategic milestone for PharmaEssentia and further strengthens the Company’s leadership position in myeloproliferative neoplasms,” said Ko-Chung Lin, Ph.D., Founder and Chief Executive Officer of PharmaEssentia. “We believe ET represents a major long-term growth opportunity for BESREMi ® and has the potential to significantly expand the reach of the Company’s hematology franchise globally. This approval also advances our broader strategy to expand the global BESREMi ® franchise as we continue preparations for a potential U.S. approval and commercial launch in ET later this year.” Dr. Lin continued, “Data generated across the Phase 3 SURPASS-ET study and the Phase 2b EXCEED-ET trial demonstrated the potential of ropeginterferon alfa-2b to achieve durable hematologic and molecular responses in ET patients, supporting the potential for durable hematologic and molecular responses consistent with disease-modifying activity in ET. We believe the differentiated pro dosing schedule of BESREMi ® position BESREMi ® as a differentiated treatment option for a broad range of ET patients.” The TFDA approval was supported by data from the global Phase 3 SURPASS-ET study, which evaluated ropeginterferon alfa-2b in high-risk ET patients resistant or intolerant to hydroxyurea. Specifically, in the global Phase 3 trial SURPASS-ET, BESREMi ® demonstrated a superior durable clinical response rate compared to anagrelide (42.9% vs. 6.0%; p=0.0001). Across clinical studies, BESREMi ® demonstrated clinically meaningful hematologic responses, molecular responses, and a manageable safety profile. In addition to the ongoing FDA review of the ET sBLA, ropeginterferon alfa-2b has been added to the National Comprehensive Cancer Network ® (NCCN ® ) Clinical Practice Guidelines in Oncology for the treatment of ET, further supporting increasing adoption of interferon-based treatment approaches in ET and other MPNs. PharmaEssentia is the inventor and owner of BESREMi ® (ropeginterferon alfa-2b-njft) and maintains intellectual property rights for the product across all indications. 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 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 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 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 Contact Muriel Huang Director, Investor Relations and Corporate Communication muriel_huang@pharmaessentia.com

百斯瑞明（Besremi，罗培干扰素α-2b-njft）是全球首款专门获批用于真性红细胞增多症（PV，真性红细胞增多症是一种罕见慢性骨髓增殖性血液病）的长效干扰素新药，也是当前全球PV治疗领域的核心创新药物，以下结合全球新药盘点信息整理其核心定位：百斯瑞明基本产品信息商品名：百斯瑞明（Besremi）通用名：罗培干扰素α-2b-njft研发企业：中国台湾药华医药（PharmaEssentia）全球获批进度：2019年率先在欧盟获批上市，2021年获美国FDA批准，是FDA近60年以来批准的首款全新机制真性红细胞增多症治疗药物；2024年正式获得中国国家药监局进口注册批准，进入中国市场。治疗定位：获批用于所有成人真性红细胞增多症患者，无论患者既往是否接受过其他治疗，都可使用。全球同类药物对比，核心创新优势突出在全球已获批的真性红细胞增多症降细胞治疗新药中，百斯瑞明的优势十分明确：唯一实现长期深度分子学缓解：相比传统一线药物羟基脲，以及JAK抑制剂芦可替尼，百斯瑞明不仅能控制血细胞计数，还可以持续降低JAK2 V617F致病突变负荷，3年治疗后53%的患者可达到完全血液学缓解，近半数患者可实现部分分子学缓解，部分患者甚至可以实现停药后长期无治疗缓解，这是现有其他PV药物暂未实现的临床获益。给药便利度大幅提升：作为新一代单聚乙二醇化长效干扰素，百斯瑞明的半衰期是传统PEG干扰素的2倍以上，维持治疗阶段仅需每月注射一次，远低于传统干扰素每周1-3次、传统PEG干扰素每两周一次的给药频率，患者可以居家自行注射，治疗依从性大幅提升。长期安全性更优：相比传统干扰素，百斯瑞明的纯度更高，副反应发生率降低了30%以上；类流感症状、严重精神副反应发生率显著降低，临床试验中因不良反应停药的比例不足8%，远低于传统干扰素。全球当前PV领域新药研发进展除百斯瑞明已成功上市外，目前全球真性红细胞增多症领域在研新药主要集中在以下方向：新一代JAK抑制剂：如fedratinib、pacritinib，目前主要针对骨髓纤维化开展研究，部分拓展至真性红细胞增多症二线治疗，暂未获批PV适应症。JAK-STAT通路精准靶向药物：如ruxolitinib透皮贴剂、JAK2特异性抑制剂SHR-1701等，目前均处于早期或中期临床试验阶段，尚未披露三期临床数据。端友酶抑制剂：如imetelstat，针对JAK抑制剂治疗失败的PV患者开展研究，目前仍处于临床研发阶段。整体来看，百斯瑞明是近十年全球真性红细胞增多症领域首款成功上市的原创新药，也推动了PV治疗从单纯控制症状向追求深度分子学缓解的理念转变，是当前全球一线治疗的首选方案之一。香港登越药业温馨提示：本文旨在介绍医药健康研究，不作任何用药依据，具体用药指引，请咨询主治医师。返回搜狐，查看更多