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FDA grants breakthrough therapy designation to ACE910 for hemophilia A
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The FDA granted breakthrough therapy designation to ACE910 for the prophylactic treatment of patients aged at least 12 years who have hemophilia A with factor VIII inhibitors, according to a press release from the drug’s manufacturer.
The FDA based its decision in part on results of a phase 1 trial presented at the 2014 ASH Annual Meeting and Exposition and the subsequent phase 1/2 extension study presented at the International Society of Thrombosis and Haemostasis 2015 Congress.
Results of the phase 1 study showed ACE910 (Genentech/Roche), administered as a weekly subcutaneous injection, appeared safe and effective for the prophylactic treatment of patients with severe hemophilia A. The cohort included patients with and without inhibitors to factor VIII, the development of which is a severe complication of hemophilia A treatment that makes it difficult to achieve a level of factor VIII sufficient to control bleeding with traditional replacement therapies, according to the release.
Genentech is planning is phase 3 trial of ACE910 in patients with hemophilia A with factor VIII inhibitors for the end of 2015, as well as a phase 3 trial of patients without inhibitors for 2016. An additional trial composed of pediatric patients is scheduled for 2016.
“People with hemophilia A may require regular and frequent infusions of replacement clotting factor to reduce the risk of dangerous bleeding, and they can develop inhibitors that make replacement ineffective," Sandra Horning, MD, chief medical officer and head of Global Product Development at Genentech, said in a press release. “We are pleased that the FDA has granted breakthrough therapy designation for ACE910, recognizing an unmet need for patients with inhibitors and the promise of these early data. Genentech has been developing antibody treatments for people with blood disorders for over 20 years, and we are excited to expedite the development of a potential new treatment for hemophilia A.”