Date: 2015-12-21
Type of information: Initiation of preclinical development
phase:
Announcement:
Company: Baxalta (USA - IL)
Product: Adynovate® - BAX 855
Action
mechanism: blood coagulation factor/protein. Adynovate®/BAX 855 is based on Advate®, a full-length FVIII molecule. Through a collaboration with Nektar Therapeutics, BAX 855 leverages proprietary PEGylation technology designed to extend the duration of activity of proteins in the body. The drug has been developed through a collaboration with Nektar Therapeutics. Adynovate® was approved by the FDA in November 2015 for use in adolescent and adult hemophilia patients (12 years and older) for on-demand treatment and control of bleeding, and prophylaxis to reduce the frequency of bleeding episodes.
Disease: hemophilia A
Therapeutic area: Rare diseases - Genetic diseases - Hematological diseases
Country:
Trial details:
Latest
news: * On December 21, 2015, Baxaltaannounced initial results from a Phase 3 clinical trial of Adynovate® [Antihemophilic Factor (Recombinant), PEGylated], an extended circulating half-life recombinant Factor VIII (rFVIII) treatment for hemophilia A based on Advat®e [Antihemophilic Factor (Recombinant)]. The prospective, uncontrolled, open-label, multi-center Phase 3 study was designed to assess the safety and immunogenicity of Adynovate®. The study enrolled 73 previously-treated patients with severe hemophilia A younger than 12 years of age and assessed the treatment’s hemostatic efficacy in prophylaxis and treatment of bleeding episodes. All participants received prophylactic Adynovate® treatment (median 1.9 infusions per week) and were followed for six months.
Adynovate® met its primary endpoint in the study, as no patients developed inhibitory antibodies to Adynovate®. In addition, no treatment-related serious adverse events were reported. More than 70 percent (72.7 percent) of patients had no joint bleeds while on treatment with Adynovate® and nearly 40 percent (37.9 percent) experienced zero bleeds. The median annualized bleeding rate (ABR) among patient participants treated with Adynovate® was 2.0 (range 0-49.8; mean ABR 3.0), which was comparable to the rates seen in the adult study.
With the study results, the company plans to file for marketing authorization in Europe and aims to file for a pediatric indication in the U.S. 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.
In addition to an ongoing study in the surgical setting, Baxalta’s continuation study remains ongoing to assess long-term safety and efficacy in previously-treated patients with severe hemophilia A. The company has also recently announced the initiation of a study of previously-untreated patients (PUPs) with severe hemophilia A, as well as a study to evaluate pharmacokinetic (PK)-guided prophylaxis dosing with previously-treated patients (the PROPEL study).
