Biotech company, Roche, has announced that the US Food and Drug Administration (FDA) has accepted its biologics licence application (BLA) and granted priority review for its once-weekly subcutaneous treatment for haemophilia A with factor VIII inhibitors.
Priority review
Emicizumab prophylaxis is an investigational bispecific monoclonal antibody that has been designed to activate the natural coagulation cascade and restore blood clotting through bringing together factors IXa and X.
“Roche has a history of developing innovative antibody therapies to address some of the highest unmet medical needs,” said Sandra Horning, MD, Roche’s chief medical officer and head of Global Product Development. “Results of our phase III study in adults and adolescents as well as early phase III results in children showed that emicizumab has significant potential to help people with haemophilia A with inhibitors, who face major challenges in preventing and treating bleeds. We are working with the FDA to hopefully bring this new prophylactic treatment option to the haemophilia A inhibitor community as soon as possible.”
The BLA for emicizumab is based on results from the phase III HAVEN 1 study in adults and adolescents 12 years of age and older, as well as interim results from the phase III HAVEN 2 study in children younger than 12 years of age. Data from both these trials have also been submitted to the European Medicines Agency (EMA) for review under accelerated assessment.
A decision from the FDA regarding an approval is anticipate in February next year.