PharmaEssentia announced on the 18th that it has submitted a new drug application in Japan for Ropeginterferon alfa-2b (Ropeg) to include an additional indication for Essential Thrombocythemia (ET). According to the review process of Japan’s Pharmaceuticals and Medical Devices Agency (PMDA), regulatory approval is expected by the third quarter of 2026, potentially benefiting around 30,000 ET patients in Japan.

The company is actively advancing global regulatory submissions for Ropeg in ET. This year, PharmaEssentia has already filed applications in China, Taiwan, and Japan, and the team is scheduled to hold a Pre-sBLA meeting with the U.S. FDA in late October to finalize submission details. The company aims to complete its U.S. application by the end of this year and secure global approvals for ET in 2026. Commercial preparations are already underway, positioning Ropeg as PharmaEssentia’s second growth engine to accelerate operational expansion.

In July, PharmaEssentia also submitted an application to the U.S. National Comprehensive Cancer Network (NCCN) to include Ropeg in its ET treatment guidelines, with the earliest potential inclusion by the end of this year. The NCCN Guidelines are globally recognized as the gold standard for cancer care, shaping clinical practice and insurance reimbursement policies. If formally recommended by the NCCN, Ropeg could be eligible for exceptional reimbursement, off-label use, or hospital procurement even prior to U.S. FDA approval—strengthening its future market access and reimbursement prospects in the ET segment.

PharmaEssentia CEO Ko-Chung Lin stated that Phase III clinical trial results have demonstrated Ropeg’s significant clinical benefits for ET patients. The company is proceeding according to plan to expand global approvals for this indication, aiming to benefit more ET patients and to further highlight Ropeg’s breakthrough therapeutic value in hematologic oncology while enhancing the international visibility of Taiwan’s innovative drug development.

Ropeg, a next-generation long-acting interferon independently developed and manufactured by PharmaEssentia, has been approved in nearly 50 countries worldwide for the treatment of Polycythemia Vera (PV), including major markets such as the European Union, the United States, and Japan. The drug continues to show steady global growth in both patient usage and sales performance.

Ropeg obtained Japanese marketing approval for PV in March 2023, was included in Japan’s National Health Insurance coverage in May, and officially launched in June 2023. Since June 2024, patients have been able to self-administer Ropeg at home. In October 2024, the company’s Japanese subsidiary further introduced home delivery services, enhancing patient convenience, treatment adherence, and overall quality of life while continuing to expand its footprint in Japan.

PharmaEssentia is also pursuing new indications for Ropeg. Both ET and PV belong to the group of myeloproliferative neoplasms (MPN) and affect a similar number of patients, making ET a natural next step following PV. According to market research, Japan has approximately 30,000 ET patients, with current standard treatments relying primarily on Hydroxyurea (HU) and Anagrelide (ANA). However, these therapies have limited efficacy and tolerability, underscoring the urgent need for innovative treatment options.

In early 2025, PharmaEssentia announced positive results from its global Phase III SURPASS ET study, which evaluated a high-dose regimen of Ropeg (250 mcg–350 mcg–500 mcg) designed to achieve target doses more rapidly. Results showed that the Ropeg group achieved a durable clinical response rate of 42.9%, compared with 6.0% in the Anagrelide group (p=0.0001), demonstrating a statistically significant and clinically meaningful advantage.

The SURPASS ET results were presented as an oral presentation at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting, where the study was selected as a “Best of ASCO” featured paper. It was also chosen as one of the Top Six Abstracts at the European Hematology Association (EHA) Annual Congress, presented in the Plenary Session—the conference’s highest honor. These dual recognitions have drawn widespread attention and strong interest from the global scientific and MPN medical communities, further solidifying Ropeg’s position as a potential new standard in ET therapy.

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