Despite setbacks, Portola snags FDA OK for Bevyxxa
- The Food and Drug Administration has approved Portola Pharmaceuticals' Bevyxxa (betrixaban) for use as an oral, once-daily anticoagulant for prophylaxis for in hospital and up to six weeks after for patients hospitalized with an acute medical illness and who are at high risk of venous thromboembolism (VTE).
- The approval for Bevyxxa, a factor Xa inhibitor, follows fast track designation and priority review, and comes a day before the Prescription Drug User Fee Act (PDUFA) action date of June 24, 2017.
- The results are based on the Phase 3 study APEX, which missed its primary endpoint by a hair but was subsequently re-analyzed by Portola
Portola is touting Bevyxxa (betrixaban) as the "first and only anticoagulant" for what is actually a rather specific indication. Yet, Bevyxxa will be entering a market with a number of established novel oral anticoagulants (NOACs).
However, the NOACs haven't captured the market against the stalwart drug warfarin in quite the way the pharma companies expected, because of perceived bleeding problems and lack of reversal agents. The big pharma backers of drugs like Eliquis (apixaban) and Xarelto (rivaroxaban) haven't sat idly by — the companies have been conducting major market pushes for the drugs in an effort to educate physicians about the benefits over the standard-of-care.
Bevyxxa has had serious challenges through its development. In 2009, Merck & Co and Portola signed an exclusive global collaboration and license agreement for BevyxXa. However, the deal only lasted two years, with Merck returning rights to a Phase 3-ready drug to Portola in March 2011, citing an ongoing prioritization of its late-stage pipeline.
In March 2017, Portola announced topline results from APEX, its Phase 3 trial, stating that the drug did not meet its primary endpoint of reduction in the number of thrombotic events. Part of the issue was a complex enrichment design for the study, which required the drug to meet the primary endpoint in patients with high risk of thrombosis.
If the drug met that endpoints, it would then be studied in intermediate-risk patients, followed by low risk patients. Because the highest-risk part of the study didn't show significance, the study was deemed a failure and terminated. At the time, the news trimmed about 30% off the value of shares in Portola. The company went ahead with submission, though, and the Food and Drug Administration accepted the new drug application for Bevyxxa in December 2016.
Further analysis of the study had suggested a simpler trial design might have shown a more positive outcome for Bevyxxa, giving the FDA a basis for approval.
Bevyxxa's marketing authorization approval has also been accepted in Europe, and a decision is expected within the coming weeks.
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