Wednesday, December 28, 2016
Shire plc announced that the U.S. Food and Drug Administration (FDA) has approved ADYNOVATE [Antihemophilic Factor (Recombinant), PEGylated], an extended circulating half-life recombinant Factor VIII (rFVIII) treatment for hemophilia A, in pediatric patients under 12 years of age. The FDA also approved ADYNOVATE for use in surgical settings for both adult and pediatric patients. 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, M.D., 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 3 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 percent (73 percent) of children had zero joint bleeds (n=48/66) while on prophylactic treatment with ADYNOVATE and nearly 40 percent (38 percent) experienced zero bleeds (n= 25/66). The median overall annualized bleeding rate (ABR) among pediatric patients treated with ADYNOVATE was 2.0 (mean ABR 3.04; range 2.21-4.19), which was similar to the rates seen in the adult study.
"At Shire, we are dedicated to developing innovative therapies for patients living with hemophilia A," said Philip J. Vickers, Ph.D., Global Head of Research and Development at Shire. "Today's 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."
Approval to use ADYNOVATE in surgical settings for both adult and pediatric patients was based on interim results of an ongoing Phase 3 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. ADYNOVATE 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. The most common adverse reactions reported in >= 1% of subjects in the clinical studies were headache and nausea. Through a collaboration with Nektar Therapeutics (NASDAQ: NKTR), ADYNOVATE leverages proprietary PEGylation technology designed to extend the amount of FVIII available for use in the body. The technology was selected because it maintains the integrity of the parent molecule (ADVATE) while reducing the time at which the body clears ADYNOVATE, resulting in an extended circulating half-life.
