FDA Approves PharmaEssentia’s Besremi for Adults with Polycythemia Vera

The U.S. Food and Drug Administration has approved Taiwan-based PharmaEssentia’s Besremi for the treatment of adults with polycythemia vera.

Polycythemia vera (PV) is a rare, chronic, and life-threatening blood cancer caused by a mutation in stem cells in the bone marrow, resulting in the overproduction of blood cells. When this occurs, it puts a person at risk for serious health problems, including blood clots, stroke and heart attack. Most cases are caused by a JAK2V617F mutation and, without proper management, this debilitating cancer can progress into myelofibrosis and malignancies, including acute myeloid leukemia.

Besremi (ropeginterferon alfa-2b-njft) is the only interferon approved for PV. It is a monopegylated, long-acting interferon that exhibits its cellular effects in polycythemia vera in the bone marrow. Besemri was approved with a boxed warning for risk of serious disorders including aggravation of neuropsychiatric, autoimmune, ischemic, and infections disorders. PharmaEssentia is preparing to make Besremi available in the coming weeks in the United States.

“The FDA approval of Besremi for people with polycythemia vera represents the next step in advancing patient care as it provides a critical addition to managing not only symptom burden and near-term complications, but also treating the cancer early, which may help reduce the risk of disease progression over time,” said Srdan Verstovsek, director of the Hanns A. Pielenz Clinical Research Center for Myeloproliferative Neoplasms, Department of Leukemia at the University of Texas MD Anderson Cancer Center.

The FDA approval was based on safety from the PEGINVERA and PROUD/ CONTINUATION-PV studies and efficacy data from the PEGINVERA clinical study program. The study showed that after 7.5 years of treatment with Besremi, 61 percent of patients with PV experienced a complete hematological response (defined as hematocrit <45 percent without phlebotomy for at least 2 months since last phlebotomy, platelets ≤ 400 x 109/L, leukocytes ≤10 x 109/L, normal spleen size (longitudinal diameter ≤12 cm for females and ≤ 13 cm for males).

Importantly, 80 percent of patients achieved a hematological response (based on objective laboratory parameters only, with the exclusion of normal spleen size and thrombosis). These parameters are the most commonly used metrics to make therapeutic decisions. In the pooled safety population of patients treated with Besremi, the most common adverse reactions (incidence greater than 40 percent) were influenza-like illness, arthralgia, fatigue, pruritis, nasopharyngitis, and musculoskeletal pain. Serious adverse reactions (incidence greater than 4 percent) were urinary tract infection, transient ischemic attack and depression.

“As we begin working closely with the community to integrate this important treatment into clinical practice, we also continue to expand our scientific efforts to unlock the full potential of our pioneering molecule,” said Ko-Chung Lin, co-founder and CEO of PharmaEssentia and inventor of Besremi.

Besremi has orphan drug designation for treatment of PV in the United States. The product was approved by the European Medicines Agency in 2019 and has received approval in Taiwan and South Korea.

Author: Rare Daily Staff