- The U.S. Food and Drug Administration turned down the Biologics License Application (BLA) for Portola Pharmaceutical's anticoagulant reversal agent, issuing a Complete Response Letter (CRL) to the company.
- Portola hosted a conference call with investors before the open of the market on Thursday, noting it had its own questions for the FDA and that it still needed clarification regarding how to resolve the CRL.
- The biotech added that the Chemistry, Manufacturing and Controls section of the BLA may not be complete and could be what is creating the delay. The company said they were hoping to sort out the details of this post-approval.
In the CRL for AndexXa, the agency requested that Portola provide additional information on manufacturing, as well as additional data to support inclusion of edoxaban and enoxaparin in the label. The letter also indicated that there needs to be further review and discussion of recently submitted clinical amendments to Portola’s post-marketing commitments.
"We do expect to be communicating with the FDA in the very near-term," said Portola CEO Bill Lis on the call. "We were confident in the science, confident in the clinical data that we provided, and confident that we met the requirements of accelerated approval. So leading up to the PDUFA date this was a little bit of a surprise."
"We are disappointed," he added.
Portola isn't the only one that is disappointed, investors and analysts showed their distaste for the development. Portola's stock opened the day down more than 10% and lost nearly another 20% within the first hour of the trading day to linger near $19 per share.
Meanwhile, commentary on Twitter from analysts showed the frustration with the company and its lack of thorough explanations for the delay.
i'd rather hear $PTLA say their dog ate the CMC section than their rationale for incompleteness...wtf?— zach (@zbiotech) August 18, 2016
Lis said the company is hoping to resolve the issues in the letter by the end of the year, but that the timeline would be uncertain until Portola had further discussions with the FDA.
AndexXa is a Factor Xa inhibitor reversal agent meant to be used in emergency situations when products like Eliquis (apixaban) need to be reversed due to uncontrolled bleeding.