The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion for the marketing authorisation Baxalta Incorporated's Obizur (antihemophilic factor (recombinant), porcine sequence).
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.
“The positive support by the European regulators reflects the value this therapy can provide to patients, and supports Baxalta’s long-standing commitment to addressing unmet needs and reducing the global burden of bleeding disorders.”
The CHMP positive opinion is based on a global, prospective, controlled, multi-center phase II/III open-label clinical trial that examined the efficacy of Obizur in the treatment of serious bleeding episodes in adults with acquired haemophilia A (29 patients evaluated for safety, 28 evaluated for efficacy). All patients treated with Obizur (28/28) showed a positive response (bleeding stopped or reduced) and clinical improvement, with FVIII activity levels at least greater than 20 per cent at 24 hours after the initial infusion. Resolution of the initial bleeding episode was observed in 24 out of 28 (86 per cent) of the patients treated with Obizur. Common adverse reactions observed in greater than five per cent of patients in the clinical trial were development of inhibitors to porcine FVIII.
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.
Obizur is not indicated for the treatment of congenital haemophilia A or von Willebrand disease. Common adverse reactions observed in greater than 5 per cent of subjects in the clinical trial were development of inhibitors to porcine factor VIII.
Acquired haemophilia A is a rare, potentially life-threatening bleeding disorder, which, unlike congenital hemophilia, typically affects older adults and occurs in both males and females. In acquired haemophilia A, individuals typically experience subcutaneous, soft tissue, and post-surgical bleeding. The comorbidities in this typically elderly population also pose a particular challenge to treat serious bleeding episodes.
Baxalta Incorporated is a $6 billion global biopharmaceutical leader developing, manufacturing and commercializing therapies for orphan diseases and underserved conditions in haematology, oncology and immunology.