The FDA has granted breakthrough therapy designation status to Genentech’s rituximab (Rituxan) for pemphigus vulgaris (PV), a rare, serious, and life-threatening autoimmune disease with limited treatment options.
The designation is intended to expedite the development and review of medications with early evidence of potential clinical benefit in serious diseases and to help ensure that patients get access to these medications as soon as possible. Patients diagnosed with PV are currently being enrolled for a phase 3 trial of rituximab.
Rituximab is currently approved for the treatment of non-Hodgkin’s lymphoma, chronic lymphocytic leukemia, rheumatoid arthritis, granulomatosis with polyangiitis, and microscopic polyangiitis.
The breakthrough status came following a review of data from a Roche-supported randomized trial conducted in France that evaluated rituximab plus oral corticosteroid treatment compared with corticosteroid as a first-line treatment in patients with moderate to severe pemphigus. (Roche markets rituximab outside the United States and Japan.) The study, which was published in The Lancet, showed that Rituxan may provide substantial improvement in disease remission rates and successful tapering and/or cessation of corticosteroid therapy.
Meanwhile, there’s been progress with developing a biosimilar for rituximab. The European Medicines Agency (EMA) is expected to soon grant approval for Celltrion’s rituximab biosimilar (Truxima). The EMA’s Committee for Medicinal Product for Human Use announced on December 16, 2016, that it had recommended granting marketing approval for rituximab biosimilar. European oncologists expect the arrival of a rituximab biosimilar to slash costs and expand patient access to the expensive oncology treatment. Europe would be the first big market for rituximab biosimilars because a US approval of biosimilar rituximab is probably farther in the future.
The rituximab biosimilar has already penetrated the Asian market—it was approved in 2016 in South Korea, covering all indications of the reference drug. In the United States, Celltrion is collaborating with Teva, which will commercialize the biosimilar if approved here.