In the race to win FDA approval for a long-acting hemophilia A treatment, Novo Nordisk ($NVO) said its factor VIII therapy came through in a Phase III study, helping the Danish drugmaker play catchup in a three-way contest with Biogen Idec ($BIIB) and Bayer.
Administered every fourth day, the investigative N8-GP (turoctocog alfa pegol) proved itself safe and effective in a Phase III trial testing its worth as a prophylaxis and an on-demand treatment for bleeds, Novo said. Like the recently approved Novoeight, N8-GP treats hemophilia A by replacing the key coagulation protein factor VIII, but the new formulation is glycopegylated to extend its half-life and allow for less frequent administration.
In a 186-patient study, those taking the drug as a prophylactic posted a median annualized bleeding rate of 1.3, and on-demand patients had 30.9 episodes per year, results that put it in the same ballpark as Biogen's Eloctate and Bayer's BAY 94-9027, its competitors in the long-acting race.
However, on the safety side, one patient in the study developed an efficacy-threatening factor VIII inhibitor, a problem not present in Biogen and Bayer's Phase III data. But the single inhibitor may not come back to haunt Novo when it takes N8-GP to the FDA, ISI analyst Mark Schoenebaum said in an investor note, as similar issues in previously treated patients haven't kept other factor VIII drugs from approval.
The latest study is one of four Phase III trials on N8-GP, including one testing out a once-weekly administration. Novo expects to finalize and report out data from the remaining three within the next 12 months, and the full results will shed light on how the drug stacks up to its rivals. As Schoenebaum notes, Biogen's drug posted a median annualized bleeding rate of 3.6 on a once-a-week dose, and Bayer's came in at 3.9.
Meanwhile, Biogen has already submitted its long-acting candidate for FDA review, enduring a three-month setback in December, and Bayer expects to do the same in the second half of next year. Novo hasn't disclosed a regulatory timeline for N8-GP, but Chief Science Officer Mads Krogsgaard Thomsen said the drug is moving right along and showing promise along the way.
"These results show that N8-GP has the potential to reduce the burden of treatment by decreasing the number of intravenous infusions while achieving strong results in terms of efficacy and safety for people with hemophilia A," Thomsen said in a statement.
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