The US Food and Drug Administration (FDA) has awarded priority review status to Roche’s emicizumab prophylaxis, a once weekly subcutaneous treatment used in conjunction with factor VIII inhibitors for patients with haemophilia A.
The decision follows the phase III HAVEN 1 study in adults and adolescents, as well as interim results from the phase III HAVEN 2 study in children younger than 12 years of age.
Sandra Horning, Roche’s chief medical officer, said: “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.”
Haemophilia affects around 320,000 people worldwide, with 50-60% of that number suffering from a ‘severe’ form of the disorder.
Horning added: “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 FDA is expected to make a decision on the pharma giant’s injection late February next year.
Meanwhile, Roche has additional trials of the drug ongoing, with and without inhibitors, to explore other, less frequent dosing regimens.