Ipsen is a pharmaceutical company based out of Paris. Sadly, they have just announced that they have pressed pause on not one but two studies for fibrodysplasia ossificans progressiva (FOP). FOP is an ultra-rare condition which causes bones to grow within the muscles. It progressively leads to disability and decreases life expectancy.

The Therapy

The reason for the pause is due to data that showed the therapy was unlikely to be successful. When Ipsen acquired the therapy, called palovarotene, they announced they were expecting to file for approval in the second half of last year. This drug is a molecule that researchers believe helps to regulate one of the proteins affecting the growth of the skeletal system.  The hope was that it could serve as a therapeutic option for episodic flare-ups for patients. Assuming approval, the company expected to launch the treatment in the middle of this year.

Now, that is unlikely to happen. However, that also doesn’t mean that they are completely giving up on this investigative therapy. The trials for this drug are still active even though dosing has been put on hold. The trials are still active because the researchers have not discounted the showcased evidence of therapeutic activity.

The pause on trials including this drug technically began in December when a partial hold was placed by the FDA for patients (FOP and multiple osteochondromas) participating in palovarotene trials who were under the age of 14. This initial hold was due to data which showed that the growth plate was closing early in young FOP patients treated with the drug.

So what is going to happen next? Ipsen is working to analyze their data and discussing recommended next steps with regulators. Hopefully, a revised version of this trial can begin soon.

Regeneron

You can read more about these investigative therapies here.