Despite an FDA rejection only three months ago, the saga of Ipsen’s rare disease candidate isn’t over yet—and there’s a chance it could still have a happy ending.
The biotech ended 2022 on a downer, with the FDA rejecting an application for palovarotene to treat an ultra-rare bone growth disorder called fibrodysplasia ossificans progressiva (FOP). The decision came two months after the agency delayed an advisory panel meeting to discuss palovarotene and requested additional data.
Now that Ipsen has sent over this information, the FDA has scheduled a decision date of August 16 for the resubmitted approval application. The extra data includes additional analyses spanning a range of trials, including MOVE, the first phase 3 study on people with FOP, Ipsen said in a release yesterday afternoon.
An interim analysis of the 107-participant MOVE study in December showed a 60% reduction in new abnormal bone formation heterotopic ossification volume in participants treated with palovarotene, compared to those on standard of care.
Not content with taking on just the FDA again, Ipsen has also requested that the European Medicines Agency re-examine the recommendation of its committee in January not to authorize palovarotene. At the time, the biotech said it would call for the decision to be reviewed.
The FDA and EMA rejections were only the latest obstacles in what has been an uphill journey for palovarotene since Ipsen acquired the drug in a $1.31 billion purchase of Clementia Pharmaceuticals in 2019. David Meek, Ipsen’s CEO at the time, called the med “largely derisked,” but subsequent developments suggest that verdict was a tad optimistic.
The drug would go on to receive a four-month partial clinical hold on a study in pediatric populations with FOP and multiple osteochondromas after evidence of cases of early growth plate closure. Dosing in a phase 3 FOP trial that included adults was also paused after early evidence suggested the trial would not meet its primary endpoint.
The company pulled its first new drug application in August 2021 to add more data, and the FDA accepted the resubmitted file in June 2022. While Ipsen hashed out its approval package in the U.S., the drug was approved in Canada in January.
Ipsen’s persistence can be explained by the fact that there remain no FDA-approved drugs for FOP. BioCryst axed development of a FOP candidate in November, leaving Ipsen and Regeneron as the two major players left in the space. Should the FDA’s decision go Ipsen’s way in August, it will still be ahead of Regeneron, which is expected to submit its approval application next year.