Date: 2011-07-26
Type of information: Results
phase: 1-2a
Announcement: results
Company: BiogenIdec (USA) Swedish Orphan Biovitrum (Sweden)
Product: recombinant factor VIII Fc fusion protein (rFVIIIFc)
Action mechanism:
Disease: Hemophilia A
Therapeutic area: Hematological diseases
Country:
Trial
details: The Phase 1/2a open-label, cross-over, multi-center, dose-escalation study evaluated the safety and pharmacokinetics of an intravenous injection of rFVIIIFc in 16 previously-treated patients with severe hemophilia A. The primary objective of the study was to assess the safety of rFVIIIFc at different doses; the secondary objective was to estimate the pharmacokinetic parameters of rFVIIIFc at doses ranging from 25 to 65 IU/kg.
Latest
news: Biogen Idec and Swedish Orphan Biovitrum have announced Phase 1/2a trial data showing that the companies\' long-lasting fully-recombinant factor VIII Fc fusion protein (rFVIIIFc) was well tolerated and demonstrated an approximately 1.7-fold increase in half-life compared with Advate® (antihemophilic factor recombinant, plasma/albumin-free method, rFVIII), a commercially-available factor VIII product, in 16 previously-treated patients with severe hemophilia A.
rFVIIIFc was well tolerated in this single-dose study, with no drug-related serious adverse events. Adverse events were observed in 11 out of 16 patients, with one related to study drug - dysguesia (abnormal taste in the mouth). There were no signs of injection site reactions, inhibitor development or anti-rFVIIIFc drug antibodies. rFVIIIFc demonstrated an approximately 1.7-fold increase in half-life compared to Advate. Other PK parameters such as mean residence time and area under the curve (AUC) were similarly increased. Furthermore, peak plasma concentration and AUC also demonstrated an increase proportional to the dose administered relative to Advate. Advate and rFVIIIFc had comparable and dose-dependent peak plasma concentration, and comparable recovery.
The findings, which were seen consistently across all patients and dose levels, are being presented today at the XXIIIrd Congress of the International Society on Thrombosis and Haemostasis in Kyoto, Japan.
Additionally, the European Medicines Agency\'s (EMA) Pediatric Committee recently adopted an opinion agreeing to the pediatric investigational plan for rFVIIIFc. In accordance with the opinion, Biogen Idec and Swedish Orphan Biovitrum plan to initiate a global pediatric trial in previously-treated patients under 12 years of age as soon as sufficient data are available from a study of older patients. Under draft guidelines published by the EMA for the development of factor VIII products, pediatric data from this trial will be required in the initial submission of a Marketing Authorization Application to the European regulatory agency.
