The FDA ( U.S. Food and Drug Administration ) has approved Adynovate [ antihemophilic factor ( recombinant), PEGylated ], an extended circulating half-life recombinant Factor VIII ( rFVIII ) treatment for hemophilia A ( congenital factor VIII deficiency ), 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 treatment for hemophilia A with more than 13 years of real-world patient experience.
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% ( 73% ) of children had zero joint bleeds ( n=48/66 ) while on prophylactic treatment with Adynovate and nearly 40% ( 38% ) 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.
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 greater than or equal to 1% of subjects in the clinical studies were headache and nausea.
The PEGylation technology was selected because it maintains the integrity of the parent molecule ( Advate ) and reduces the time at which the body clears Adynovate, resulting in an extended circulating half-life. ( Xagena2016 )
Source: Shire, 2016