PharmaEssentia announced on October 31 that it has officially submitted a New Drug Application (NDA) to the U.S. FDA for Ropeginterferon alfa-2b (Ropeg) to treat primary essential thrombocythemia (ET), aiming for approval in 2026. The company plans to target the U.S. ET patient market of approximately 150,000 individuals, positioning Ropeg as its second growth engine.

CEO Ko-Chung Lin stated, “Results from Ropeg’s Phase III clinical trial for ET have clearly demonstrated significant clinical benefits for patients. PharmaEssentia will continue to advance its global regulatory strategy, with additional marketing authorizations expected next year, showcasing Ropeg’s breakthrough clinical value in hematologic malignancies, benefiting more ET patients and further enhancing the international visibility of Taiwanese innovative medicines.”

Ropeg was granted U.S. orphan drug designation for ET in 2014. This NDA submission for ET includes a request for Priority Review, highlighting Ropeg’s potential superiority over existing treatments and its ability to meet urgent medical needs, which could accelerate the FDA review timeline and enable patients to access the therapy sooner.

Under FDA regulations, the agency is expected to notify the company within approximately 60 days whether the application is complete and ready for substantive review.

Ropeg is a next-generation, long-acting interferon invented and manufactured by PharmaEssentia. It is already approved in roughly 50 countries worldwide for the treatment of polycythemia vera (PV), including major markets such as the EU, the U.S., and Japan, with a steadily growing patient base and sales.

PharmaEssentia is actively expanding Ropeg’s indications. ET and PV are both myeloproliferative neoplasms (MPNs) with similar patient populations. Market research indicates around 150,000 ET patients in the U.S., where the primary treatments are hydroxyurea (HU) and anagrelide (ANA), both of which have limited efficacy and tolerability, highlighting the urgent need for innovative therapies for ET.

The company expects to receive U.S. approval for ET in 2026, allowing Ropeg to expand from PV to the ET treatment market, serving as a second engine for growth. Additionally, the global Phase III trial of Ropeg for early-stage myelofibrosis (HOPE-PMF) is actively progressing. With the steady advancement of global clinical trials and regulatory strategies, PharmaEssentia is steadily moving toward becoming a leading international innovator in the MPN field.

Ko-Chung Lin added, “We have fully planned our entry into the ET market. In the U.S., sales, marketing, medical affairs, and market access strategies have already begun. Our sales team expanded by 65% mid-year to support ongoing PV sales and prepare for ET market entry. Medically, we have applied to include Ropeg as a recommended treatment option for ET patients in the U.S. National Comprehensive Cancer Network (NCCN) guidelines, aiming to broaden patient access. For market access, we have initiated pre-launch discussions and strategic planning with insurers. Normally, market access preparations take over a year, but because this is an additional indication, the timeline can be significantly shortened.”

PharmaEssentia has accelerated its global ET regulatory submissions. This year, it has filed for Ropeg ET approval in China, Taiwan, and Japan, and completed communication with the U.S. FDA in September. With complete data and a clear review plan, the FDA confirmed that the company may proceed directly to submission without a pre-NDA meeting. The FDA also acknowledged that Ropeg’s global Phase III “SURPASS ET” trial serves as the pivotal study, supported by U.S. Phase II “EXCEED ET” clinical data as confirmatory evidence, sufficiently demonstrating Ropeg’s efficacy and safety in U.S. ET patients.

Resource: 藥華藥Ropeg申請美國ET藥證 拚2026取證