Biogen Submits Hemophilia B BLA - Analyst Blog

Biogen Idec (BIIB) recently submitted a Biologics License Application (BLA) seeking US Food and Drug Administration (FDA) approval for its long-acting factor IX (rFIXFc) candidate for hemophilia B.

Biogen and its partner, Swedish Orphan Biovitrum, had presented positive phase III top-line results on rFIXFc last year. 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.

Meanwhile, Biogen intends to file for FDA approval of its hemophilia A candidate, rFVIIIFc, as well in the first half of 2013.

A convenient dosing schedule (supported by a longer duration of action and a suitable safety profile) could help rFIXFc and rFVIIIFc capture share from existing products in the hemophilia market.

Upcoming Catalysts

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. A response in the US should be out by March. We believe BG-12 could become a leader in the oral multiple sclerosis market once launched.

Another important near-term event is the presentation of top-line phase III (ADVANCE) results on multiple sclerosis candidate, PEGylated interferon.

Biotech companies that currently look interesting include Amgen (AMGN), which carries a Zacks #1 Rank (Strong Buy).