The US Food and Drug Administration approved ropeginterferon alfa-2b-njft (BESREMi) for the treatment of adults with essential thrombocythemia, regardless of genotype or disease status, according to a press release from PharmaEssentia USA Corporation. The indication includes newly diagnosed patients who have not received cytoreductive therapy.
The approval marks the first new FDA-approved treatment for essential thrombocythemia (ET) in nearly 30 years and expands the indication for ropeginterferon alfa-2b-njft, which is also approved for adults with polycythemia vera.
ET is a chronic blood cancer and a type of myeloproliferative neoplasm characterized by overproduction of platelets in the bone marrow. Patients with ET are at increased risk of blood clots, abnormal bleeding, and an enlarged spleen. Complications can include heart attack, stroke, and pulmonary embolism.
The FDA based the approval on data from the global phase 3 SURPASS ET trial. According to PharmaEssentia, durable response rates were higher with ropeginterferon alfa-2b-njft than with anagrelide, and treatment reduced thromboembolic events over 12 months. The data also showed durable responses based on modified European Leukemia Net criteria and sustained hematologic control in adults with ET.
Ropeginterferon alfa-2b-njft is a long-acting interferon-based therapy developed with monopegylation technology. PharmaEssentia stated that the treatment is designed to target disease-driving bone marrow cells and is intended to lower platelet counts and disease burden.
The prescribing information includes a boxed warning concerning potentially fatal neuropsychiatric, autoimmune, ischemic, and infectious disorders associated with interferon alfa products. Contraindications include a history or presence of severe depression, suicidal ideation, or suicide attempt; hypersensitivity to interferons or inactive ingredients; moderate or severe hepatic impairment; a history or presence of active serious or untreated autoimmune disease; and a history of transplantation in patients receiving immunosuppressive agents.
“For nearly 30 years, people living with ET and the physicians treating them have had limited treatment innovation,” said Ruben Mesa, MD, principal investigator of SURPASS ET and president of Advocate Health’s Cancer National Service Line. “In my experience, patients need treatment options that not only control blood counts but also address the underlying disease.”
PharmaEssentia said ropeginterferon alfa-2b-njft was expected to be available in the US immediately following approval.
Source: PharmaEssentia USA Corporation
