Portola Pharmaceuticals ($PTLA) had expected to launch its first drug this year. But those plans have been pushed back as its lead candidate Andexxa, developed to reverse anticoagulation for Factor Xa inhibitors, has received a complete response letter from the FDA.
The company said on a conference call that it could resubmit Andexxa to the FDA before year-end. Portola shares were down about one-third, pushing it to a market cap of about $1.1 billion.
The agency asked for Portola to offer additional information, primarily related to manufacturing. It also asked for more data to support the inclusion of edoxaban and enoxaparin in the label; these are specific Factor Xa inhibitors, with the others named in its planned indication being apixaban and rivaroxaban.
Two Phase III trials evaluated Andexxa’s reversal of the anticoagulants rivaroxaban and apixaban in older healthy volunteers, with the data published in The New England Journal of Medicine last November.
The FDA also said it needs to review clinical amendments that Portola recently submitted on its post-marketing commitments, the company said.
Portola hopes Andexxa will become the first universal antidote for Factor Xa inhibitors.
“Because Andexxa addresses an urgent unmet medical need, we and the FDA are committed to resolving the outstanding questions and determining appropriate next steps,” said the company’s CEO Bill Lis in a statement. “Portola’s goal is to define the most expedient path to approval so we can meet the needs of these patients who have no alternative.”
Andexxa has both an orphan drug and a breakthrough therapy designations from the FDA.
Up next, Portola has betrixaban, which is an oral, once-daily Factor Xa inhibitor anticoagulant. It’s in Phase III testing to treat venous thromboembolism in acute medically ill patients. Betrixaban has fast track designation from the FDA and Portola hopes to launch it next year--despite the fact that it failed in pivotal testing.
- here is the release
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