Dive Brief:
- An group of advisers to the Food and Drug Administration on Wednesday voted in favor of an experimental drug Ipsen Biopharmaceuticals has been developing for a rare bone disease known as fibrodysplasia ossificans progressiva.
- After an initial rejection last year, Ipsen submitted a new request that included new findings from an after-the-fact study analysis. The advisory committee voted 10-4 that the data show the drug is effective and 11-3 that the benefits of therapy outweigh the risks.
- Fibrodysplasia ossificans progressiva, or FOP, is an extremely rare and degenerative condition that causes muscle and connective tissue to turn into bone. The disease, estimated to affect less than 1,000 people worldwide, has no available treatments that can change its course. If approved by the FDA, Ipsen’s drug would be the first in the U.S.
Dive Insight:
Ipsen’s drug, palovarotene, has been in clinical development for several years and changed hands multiple times.
The medicine was originally developed by Roche, but failed in clinical testing as a potential treatment for chronic obstructive pulmonary disease.
It wasn’t until Canadian biotechnology Clementia Pharmaceuticals licensed the drug in 2014 that palovarotene became a potential treatment for FOP. Clementia was studying its effects on bone formation, and made enough progress that Ipsen agreed to acquire the company for $1.3 billion in 2019 to boost its rare disease work.
But the drug has had a bumpy path to approval. The drug delivered mixed results in testing and was submitted to the FDA multiple times. The regulator rejected it in December and asked for more data, leading Ipsen filed a new application in March and add more analyses of the results.
Even then, the FDA had some reservations. The data showed signs that patients taking the drug might be more likely than placebo recipients to experience the inflammatory “flare-ups” that come before abnormal bone growth, according to documents filed ahead of the meeting. Staff reviewers also questioned the drug’s effectiveness.
Despite those concerns, a majority fo panelists expressed support. While the FDA doesn’t always follow the advice of its advisory panels, it typically does.
A decision is expected by August 16.
The drug is currently available for use in Canada and the United Arab Emirates, where it’s sold as Sohonos.