Baxalta announced initial results from a Phase 3 clinical trial of Adynovate [Antihemophilic Factor (Recombinant), PEGylated] for the treatment of hemophilia A in patients <12 years old. Adynovate was recently approved by the Food and Drug Administration (FDA) in November 2015 for use in hemophilia patients ≥12 years old for on-demand treatment and control of bleeding, and prophylaxis to reduce the frequency of bleeding episodes.
The prospective, uncontrolled, open-label, multicenter Phase 3 study assessed the hemostatic efficacy in prophylaxis and treatment of bleeding episodes, the safety and the immunogenicity of Adynovate in 73 previously-treated patients (PTPs) <12 years old with severe hemophilia A. All subjects received prophylactic Adynovate (median 1.9 infusions per week) and were followed for 6 months.
Preliminary results showed that the study met its primary endpoint, as no patients developed inhibitory antibodies to Adynovate. No treatment-related serious adverse events were reported, showing that 72.7% of Adynovate-treated patients experienced no joint bleeds while 37.9% had zero bleeds. Study participants treated with Adynovate had a median annualized bleeding rate (ABR) of 2 (range 0-49.8; mean ABR 3), which was comparable to the rates in the adult study. Positive results from this study will support the company’s application to file for a pediatric indication in early 2016.
Baxalta initiated additional clinical trials for Adynovate, including studies on long-term safety and efficacy in PTPs with severe hemophilia A, on previously-untreated patients (PUPs) with severe hemophilia A, and on pharmacokinetic (PK)-guided prophylaxis dosing (PROPEL study).
Adynovate is an extended circulating half-life recombinant Factor VIII (rFVIII) based on Advate [Antihemophilic Factor (Recombinant)]. It uses a proprietary PEGylation technology designed to extend the amount of FVIII available in the body by maintaining the integrity of the parent molecule (Advate) and reducing the time of its clearance, resulting in increased circulating half-life.
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