FDA Approves New Treatment for Essential Thrombocythemia

The Food and Drug Administration approved ropeginterferon alfa-2b-njft (Besremi, PharmaEssentia) for adults with essential thrombocythemia (ET) on Monday. It is the first new FDA-approved therapy for ET in nearly three decades.

Ropeginterferon alfa-2b-njft is a long-acting interferon administered by subcutaneous injection, titrated to a maintenance dose given every two weeks.

FDA based its approval on the phase 3 SURPASS-ET trial, an open-label, multicenter, randomized study that compared ropeginterferon alfa-2b-njft with anagrelide in 174 adults with ET who had an inadequate response or intolerance to hydroxyurea. At both nine and 12 months, 37.4% of patients receiving ropeginterferon alfa-2b-njft achieved a durable modified European LeukemiaNet (ELN) response, compared with 3.6% of those receiving anagrelide.

The most common adverse reactions included transaminase elevations, anemia, fever, bacterial infection, pruritus and weight loss.