An antihemophilic factor that was recently approved in the US has met the primary endpoint of a phase 3 study, according to the product’s maker, Baxalta.
The product is Adynovate, a recombinant pegylated factor VIII (FVIII) treatment based on the full-length Advate molecule.
The study enrolled patients younger than 12 years of age who had severe hemophilia A. And, thus far, none of these patients has developed inhibitors to Adynovate, which was the study’s primary endpoint.
Adynovate also proved effective for preventing bleeds in some patients and did not appear to produce any serious adverse events.
Adynovate was approved in the US last month for use as routine prophylaxis and on-demand treatment and control of bleeding episodes in hemophilia A patients age 12 and older.
Baxalta said the phase 3 data will form the basis of a US filing for a pediatric indication in early 2016.
The study was designed to assess the safety and immunogenicity of Adynovate in young, previously treated patients with severe hemophilia A. The study enrolled 73 such patients.
Researchers assessed the hemostatic efficacy of Adynovate as prophylaxis and for treatment of bleeding episodes. All patients received prophylactic Adynovate treatment (a median of 1.9 infusions per week) and were followed for 6 months.
The study’s primary endpoint was met, as no patients developed inhibitory antibodies to the drug. In addition, no treatment-related serious adverse events were reported.
About 73% of patients had no joint bleeds while on treatment, and about 38% had no bleeds of any kind.
The median annualized bleeding rate among patients treated with Adynovate was 2.0 (range, 0-49.8), and the mean was 3.0, which is comparable to the rates seen in adults treated with the product.
“These initial efficacy and safety findings indicate a potentially valuable role for Adynovate to treat pediatric patients with hemophilia A, with data consistent with what was reported in clinical studies among adult patients,” said John Orloff, MD, head of research & development and chief scientific officer at Baxalta.
With these results, the company plans to file for marketing authorization in Europe and aims to file for a pediatric indication in the US in early 2016.
Adynovate is currently under regulatory review in Japan, Canada, and Switzerland. Baxalta plans to present the complete data from this study at a congress in 2016.
