Genentech Haemophilia therapy gets FDA breakthrough designation
pharmafile | September 4, 2015 | News story | Research and Development, Sales and Marketing | Genentech, Roche, haemophilia a
Roche subsidiary Genentech has been granted FDA breakthrough therapy designation for its ACE910 (RG6013, RO5534262) haemophilia A therapy.
The designation is for the prophylactic treatment of people aged 12 years or older with haemophilia A with factor VIII inhibitors.
The breakthrough therapy designation was granted based on results of a Phase I study of ACE910 in people with severe haemophilia A, presented at the annual meeting of the American Society of Hematology (ASH) in 2014, and the Phase I/II extension study of the same patients presented at the annual meeting of the International Society of Thrombosis and Haemostasis (ISTH) in 2015.
Haemophilia A, a rare genetic disorder, occurs due to insufficient levels of the essential blood clotting protein known as factor VIII, or if the protein is defective.
People with severe haemophilia A can be susceptible to uncontrolled or difficult-to-control bleeding, including internal bleeding, especially into the joints, which can lead to the need for joint replacements.
In the Phase I study, ACE910 showed promising results as a preventative treatment administered as a weekly subcutaneous injection in people with severe haemophilia A, with and without inhibitors to factor VIII.
The development of inhibitors is a serious complication of haemophilia A treatment regardless of disease severity, making it difficult, if not impossible, to achieve a level of factor VIII sufficient to control bleeding with traditional replacement therapies.
Management of bleeding in people with haemophilia A who have inhibitors to factor VIII is a major challenge, and there remains a need for additional treatment options for such patients.
“People with haemophilia 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,” says Sandra Horning, chief medical officer and head of Global Product Development.
“We are pleased that the FDA has granted breakthrough therapy designation for ACE910, recognising 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 haemophilia A.”
Genentech said it is set to begin a Phase III trial of ACE910 in haemophilia A patients with factor VIII inhibitors by the end of 2015 and a Phase III trial in patients without inhibitors in 2016. A trial in paediatric patients with haemophilia A is also planned for 2016.
Joel Levy
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