) 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
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
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
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
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
) 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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