The FDA has approved PharmaEssentia’s BESREMi, or ropeginterferon alfa-2b-njft, for adults with essential thrombocythemia, a rare chronic blood cancer marked by excessive platelet production.
The decision makes BESREMi the first new FDA-approved treatment for essential thrombocythemia, or ET, in nearly three decades, PharmaEssentia said. The medicine is approved regardless of patients’ disease status or genotype, including for newly diagnosed people who have not previously received cytoreductive therapy.
BESREMi was already marketed in the United States for adults with polycythemia vera, another myeloproliferative neoplasm. The ET approval expands the drug’s label and adds a new option for a patient population that often requires years of disease management.
ET results from bone-marrow overproduction of platelets and can be associated with genetic mutations, including JAK2. Patients face elevated risks of blood clots, abnormal bleeding and enlarged spleen. Serious complications can include heart attacks, strokes and pulmonary embolism.
BESREMi is a long-acting interferon-based treatment designed to provide sustained disease management. PharmaEssentia said its monopegylation technology and extended half-life are intended to target disease-driving cells in bone marrow, reduce elevated platelet counts and lessen disease burden. Existing therapies generally focus on platelet counts and symptoms, with limited effect on underlying disease biology, the company said.
The FDA based its decision on data from the global Phase III SURPASS ET study, which compared BESREMi with anagrelide. The trial showed superior durable modified European Leukemia Net responses and durable hematologic control for BESREMi, according to PharmaEssentia. The treatment also reduced thromboembolic events over 12 months.
Ruben Mesa, principal investigator of the SURPASS-ET trial and president of Advocate Health’s Cancer National Service Line, said patients need options that can address the underlying disease in addition to controlling blood counts.
PharmaEssentia founder and Chief Executive Ko-Chung Lin said the approval represents a significant advance for people with ET and reflects the company’s work in myeloproliferative neoplasms.
BESREMi is expected to be immediately available in the United States. The therapy recently received regulatory approval for ET in Japan and Taiwan.
The product carries a boxed warning that interferon alfa therapies may cause or aggravate serious neuropsychiatric, autoimmune, ischemic and infectious disorders. It is contraindicated in patients with, among other conditions, severe depression or suicidal history, moderate or severe liver impairment, serious active or untreated autoimmune disease, or a history of transplantation while receiving immunosuppressive therapy.
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