PharmaEssentia Announces Taiwan Approval of BESREMi® for Essential Thrombocythemia, Marking First Global Approval in ET

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 profile and 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.