FDA Grants Breakthrough Therapy Designation to Hemophilia A Drug

Prophylactic treatment administered as a weekly subcutaneous injection for severe hemophilia A with and without inhibitors to factor VIII.

Prophylactic treatment administered as a weekly subcutaneous injection for severe hemophilia A with and without inhibitors to factor VIII.

Genentech recently announced the FDA granted Breakthrough Therapy designation to a prophylactic treatment for people who are 12 years or older with hemophilia A with factor VIII inhibitors.

Hemophilia A is a rare blood disorder which occurs when an essential blood clotting protein called factor VIII is either not present in sufficient amounts or defective. People with severe hemophilia A can undergo uncontrolled or difficult to control bleeding, including internal bleeding in the joints that can lead to joint replacement need.

In a phase 1 study, the drug ACE910 showed promising results as a prophylactic treatment administered as a weekly subcutaneous injection in people with severe hemophilia A with and without inhibitors to factor VIII. Traditional replacement therapies are not sufficient enough to regain control of factor VIII levels to control bleeding in patients with hemophilia A that have developed inhibitors.

Additional treatment options are needed for these patients to improve management of their disease.

“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,” said Sandra Horning, MD, chief medical officer and head of Global Product Development. “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.”

A phase 3 trial of ACE910 is set to take place by the end of 2015 in patients with hemophilia A with factor VIII inhibitors. A phase 3 trial in patients without inhibitors is also set to take place in 2016, in addition to a trial in pediatric patients with hemophilia A.