Biogen Idec (BIIB) and partner, Swedish Orphan Biovitrum, recently presented positive phase III top-line results on their long-acting factor IX (rFIXFc) candidate for hemophilia B.
Results from the global, open-label, multi-center B-LONG study showed that rFIXFc was effective in controlling and preventing bleeding, routine prophylaxis, and perioperative management. Results showed that a single injection of rFIXFc helped control 90.4% of bleeding episodes.
A pharmacokinetic (:PK) analysis was also conducted. The approximate terminal half-life of rFIXFc was found to be 82 hours compared to 34 hours for Pfizer’s (PFE) BeneFIX.
The candidate was also found to be well-tolerated with no inhibitors to rFIXFc being detected or cases of anaphylaxis (severe allergic reaction) being reported. Patients in the study had all switched from marketed factor IX products.
Biogen and Swedish Orphan Biovitrum are currently conducting a further analysis of the data which will be presented at a scientific meeting.
BLA Submission in First Half of 2013
With these positive results in hand, Biogen and Swedish Orphan Biovitrum intend to seek US Food and Drug Administration (:FDA) approval in the first half of 2013. A Biologics License Application (:BLA) will be submitted to the agency for the candidate.
As far as the EU is concerned, Biogen and Swedish Orphan Biovitrum are evaluating rFIXFc in children below 12 years of age.
Another hemophilia candidate, rFVIIIFc (long-acting factor VIII), is also being evaluated in children. Last year, Biogen and Swedish Orphan Biovitrum had received opinions from the EMA’s Pediatric Committee (PDCO) agreeing to the companies’ plans to conduct pediatric studies with rFIXFc and rFVIIIFc.
The companies will file for EU approval once the Kids B-LONG study is completed. According to information on the clinicaltrials site, the Kids B-LONG study is currently expected to complete in Feb 2015.
Both rFVIIIFc and rFVIXFc enjoy orphan drug and fast track status in the US as well as the EU. A convenient dosing schedule (supported by a longer duration of action and a suitable safety profile) could help these candidates capture share from existing products in the hemophilia market.
Biogen remains on track to report phase III results on rFVIIIFc (hemophilia A) from the A-LONG study by year end.
Meanwhile, Biogen has some other significant pipeline catalysts coming up, the most important being an update on the regulatory status of BG-12. BG-12, Biogen’s oral multiple sclerosis candidate, is currently under review in both the US and the EU.
The FDA has granted standard review to the candidate, which means a response should be out in late 2012/early 2013. Biogen has been facing additional competition in the multiple sclerosis market in the form of Novartis’ (NVS) Gilenya. Moreover, several companies besides Biogen are working on bringing an oral multiple sclerosis drug to market. We believe BG-12 could become a leader in the oral multiple sclerosis market once launched. BG-12 should help drive long-term growth.
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