FDA approves Shire's Adynovate

New FDA-approved indications for Adynovate provides more hemophilia A patients access to proven prophylaxis with a simple, twice-weekly dosing schedule.

Adynovate is built on the full-length Advate [Antihemophilic Factor (Recombinant)] molecule, a market leading treatment for hemophilia A with more than 13 years of real-world patient experience.

Physicians need additional treatment options for pediatric patients and those undergoing surgery with hemophilia A,” said lead investigator Eric Mullins, Research Director, Hemophilia Treatment Center, Cincinnati Children’s Hospital Medical Center. “As someone who has routinely seen the benefits of factor replacement in adult patients with hemophilia A, I am pleased to be able to provide Adynovate to more patients.”

The approval of Adynovate to treat children under the age of 12 was based on data from a prospective, uncontrolled, open-label, multi-center Phase III trial designed to assess the immunogenicity along with the safety and efficacy of Adynovate. Results from the study showed that Adynovate met its primary endpoint with no previously treated children having developed inhibitory antibodies to Adynovate. In addition, no treatment-related serious adverse events were reported. More than 70% of children had zero joint bleeds while on prophylactic treatment with Adynovate and nearly 40% experienced zero bleeds. The median overall annualized bleeding rate (ABR) among pediatric patients treated with Adynovate was similar to the rates seen in the adult study.

“This announcement of the new FDA-approved indications for Adynovate in pediatric patients less than 12 years of age and those undergoing surgery underscores our commitment to provide treatment options with proven efficacy profiles to more patients living with hemophilia A,” said Philip J. Vickers, Global Head of R&D at Shire.

Approval to use Adynovate in surgical settings for both adult and pediatric patients was based on interim results of an ongoing Phase III study of perioperative control of hemostasis among 15 patients with severe hemophilia A undergoing surgical procedures

Adynovate was first approved by the FDA in November 2015. It provides proven prophylaxis with simple, twice-weekly dosing for children and adult patients with hemophilia A, helping to further individualize options for this patient population.