CHMP adopts positive opinion of Obizur for haemophilia A

The Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for the marketing authorization of Baxalta’s Obizur.

Baxalta is seeking market authorisation in Europe of Obizur for the treatment of bleeding episodes in adult patients with acquired haemophilia caused by antibodies to Factor VIII (FVIII), a very rare and potentially life-threatening acute bleeding disorder. Following this positive opinion, the European Commission is expected to make a decision on the application later this year. Upon approval in Europe, Obizur will be the first recombinant porcine FVIII treatment available for acquired haemophilia A, allowing physicians to monitor treatment response by measuring FVIII activity levels in addition to clinical assessments.

“The marketing authorisation anticipated later this year for Obizur will be an important milestone, offering patients with acquired haemophilia A in Europe a treatment option that transforms their care by allowing physicians to monitor treatment response,” said John Orloff, M.D., head of Research & Development and chief scientific officer, Baxalta.

Obizur showed a positive response in all patients treated with it in a Phase II/III trial

The CHMP positive opinion is based on a Phase II/III clinical trial that examined the efficacy of Obizur in the treatment of serious bleeding episodes in adults with acquired haemophilia A. All patients treated with Obizur showed a positive response (bleeding stopped or reduced) and clinical improvement, with FVIII activity levels at least greater than 20% at 24 hours after the initial infusion. Resolution of the initial bleeding episode was observed in 86% of the patients treated with Obizur.

Obizur is currently approved in the United States and is under regulatory review in Canada, Switzerland, Australia and Colombia. Obizur was granted orphan-drug designation by the European Commission based on the classification of acquired haemophilia A as a rare disease and the potential for the treatment to address an important unmet medical need.