The end of year approval rush continues at the FDA. The agency has approved Shire’s Adynovate [Antihemophilic Factor (Recombinant), PEGylated] for children under 12 with hemophilia A.
The FDA also approved Adynovate for use in surgical settings for both adult and pediatric patients.
Adynovate was previously approved for prophylactic and on-demand treatment of patients 12 years and older with hemophilia A.
Safety and Efficacy
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.
While taking prophylactic treatment with Adynovate, 73% (n=48/66) of the children had zero joint bleeds while on and 38% (n= 25/66) had zero bleeds.
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).
No treatment-related serious adverse events were reported and no children developed inhibitory antibodies to Adynovate.
Approval of Adynovate in surgical settings 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. Currently, no data is available to share.
In a press release, Eric Mullins, MD, Research Director, Hemophilia Treatment Center, Cincinnati Children's Hospital Medical Center said, "Physicians need additional treatment options for pediatric patients and those undergoing surgery with hemophilia A. Dr Mullins added, "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."
Hemophilia A is a rare, chronic, genetic disorder that results in impaired clotting mechanisms due to missing or reduced levels of factor VIII. People with hemophilia A experience recurrent and extended bleeding episodes that cause pain and irreversible joint damage. Some of these bleeding episodes can be life threatening. There are an estimated 16,000 – 20,000 people in the United States with hemophilia A (and 142,000 world wide).