The US Food and Drug Administration (FDA) has approved Adynovate, a recombinant pegylated factor VIII (FVIII) product, for use in patients age 12 and older with hemophilia A.
The product can be used as routine prophylaxis and for on-demand treatment and control of bleeding episodes.
Adynovate will be available in the US in the coming weeks, according to Baxalta US Inc., the company developing the product.
Adynovate (formerly BAX 855) is built on the full-length Advate, a recombinant antihemophilic factor product that was approved by the FDA in 2003.
Adynovate consists of the full-length FVIII molecule linked to other molecules, known as polyethylene glycol (pegylated). This link extends the circulating half-life of the product and therefore extends the time between treatments.
So patients on Adynovate can receive twice-weekly doses rather than the 3 to 4 weekly doses typically required with other full-length FVIII products.
“The approval of Adynovate provides an important therapeutic option for use in the care of patients with hemophilia A and reduces the frequency of FVIII infusions needed to avoid bleeding,” said Karen Midthun, MD, director of the FDA’s Center for Biologics Evaluation and Research.
The FDA approved Adynovate based on results of a phase 2/3 trial. The study included 137 previously treated hemophilia A patients who were 12 to 65 years of age.
Patients were assigned to either twice-weekly prophylaxis (40-50 IU/kg, n=120) or on-demand treatment (10-60 IU/kg, n=17) with Adynovate.
Patients in the twice-weekly prophylaxis arm had far fewer annual bleeds than patients treated on-demand. The median annual bleed rates were 1.9 and 41.5, respectively.
Nearly all (96%) bleeding episodes (n=591) were controlled with 1 or 2 infusions of Adynovate.
None of the patients developed inhibitors to the treatment. However, there were 171 adverse events in the 73 patients who received Adynovate for about 6 months.
There were 7 events (occurring in 6 patients) that were considered possibly related to Adynovate. These included diarrhea, nausea, headache, and flushing.
Studies of Adynovate are ongoing in previously treated patients with severe hemophilia A undergoing surgery and in previously treated patients with severe hemophilia A who are under the age of 12. Baxalta is also planning to initiate a study in previously untreated patients with severe hemophilia A.
The company has filed for regulatory approval of Adynovate in Japan and expects to file for marketing authorization in Europe once the pediatric study is complete.
