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FDA grants two indications to Adynovate for hemophilia A
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The FDA approved antihemophilic factor (recombinant), PEGylated to treat hemophilia A in pediatric patients aged younger than 12 years and for use in surgical settings for adults and children, according to the drug’s manufacturer.
Antihemophilic factor (recombinant), PEGylated (Adynovate, Shire) — an extended circulating half-life recombinant Factor VIII treatment — is approved for twice-weekly dosing.
A prospective, uncontrolled, open-label, multicenter phase 3 trial — designed to evaluate the immunogenicity, safety and efficacy of Adynovate in children aged younger than 12 years — met its primary endpoint of no inhibitory antibody development in previously treated children.
No treatment-related serious adverse events were reported.
Overall, 73% of children experienced no joint bleeds and 38% experienced no bleeds while on prophylactic treatment.
Patients also demonstrated an overall annualized bleeding rate (ABR) of 2 (mean ABR = 3.04; 95% CI, 2.21-4.19), which was similar to rates observed in adults.
In addition, Adynovate was approved for use in surgical settings based on interim results of an ongoing phase 3 study to evaluate the use of Adynovate as to control hemostasis in 15 patients with severe hemophilia A undergoing surgical procedures.
The most common adverse events in both studies included headaches and nausea.
“Physicians need additional treatment options for pediatric patients and those undergoing surgery with hemophilia A,” lead investigator Eric Mullins, MD, research director of the Hemophilia Treatment Center at Cincinnati Children’s Hospital Medical Center, said in a press release. “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 antihemophilic factor (recombinant), PEGylated to more patients.”
Adynovate is currently indicated for the on-demand treatment and control of bleeding episodes, perioperative management and routine prophylaxis to reduce the frequency of bleeding episodes. The agent is built on the full-length antihemophilic factor (recombinant) molecule (Advate, Shire), which has been used to treat hemophilia A for more than 13 years.