On the evening of May 21, PharmaEssentia announced its decision to proactively withdraw its application to the China National Medical Products Administration (NMPA) for the Phase III clinical trial of P1101 for early-stage primary myelofibrosis (PMF) or low to intermediate-1 risk primary myelofibrosis. This decision follows further recommendations from the U.S. FDA to modify the primary efficacy endpoints of the trial.
This Phase III clinical trial is a multinational, multicenter study. PharmaEssentia submitted the clinical trial plan to the U.S. FDA on January 17, 2024, and had revised the plan according to FDA's initial recommendations. However, the FDA recently suggested additional modifications to the primary efficacy endpoints, prompting PharmaEssentia to voluntarily withdraw the application from the NMPA temporarily. The company plans to resubmit the application after completing the necessary revisions.
PharmaEssentia explained that primary myelofibrosis (PMF) is a type of myelofibrosis (MF) and belongs to the category of myeloproliferative neoplasms (MPNs), which also includes polycythemia vera (PV) and essential thrombocythemia (ET). According to Orphanet, a website providing information on rare diseases, there are approximately 1 to 9 PMF patients per 100,000 people worldwide. For low-risk MF patients who exhibit symptoms, current treatment options include off-label use of hydroxyurea (HU), long-acting interferons, JAK2 inhibitors in some cases, or bone marrow transplantation, with JAK2 inhibitors primarily used for intermediate to high-risk MF patients.
PharmaEssentia highlighted that Ropeginterferon alfa-2b is a next-generation, long-acting interferon developed and produced in-house. It has been approved for use in adults with polycythemia vera (PV) in nearly 40 countries worldwide, including major new drug markets such as the European Union, the United States, and Japan.
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