Dive Brief:

• After years of fits and starts, Ipsen won Food and Drug Administration approval to sell the first treatment for a rare disease known as fibrodysplasia ossificans progressiva, or FOP.

• The FDA cleared the drug, palovarotene, for females who are least 8 years old and males at least 10 years old, Ipsen said Wednesday. It’s designed to reduce the formation of bone in tissue that is characteristic of the debilitating condition.

• Ipsen will sell the medicine under the brand name Sohonos at an average list price of $624,000 a year, though the cost will vary depending on insurance coverage, patient needs and support programs. A company spokeswoman said Ipsen is committed to making the treatment accessible to the approximately 400 people in the U.S. who have the disease.

Dive Insight:

The road to the U.S. market has been a winding one for Sohonos and Ipsen. Roche originally developed the medicine as a treatment for chronic obstructive pulmonary disease, but it failed in testing. The Canadian biotech Clementia Pharmaceuticals then licensed the medicine and in 2014 won orphan drug and fast track designations from the FDA to help speed development for FOP.

The initial studies went well enough to attract the attention of Ipsen, which agreed in 2019 to buy Clementia for as much as $1.3 billion. But clinical trials delivered mixed results for Ipsen, and the FDA rejected the drug in late 2022. Ipsen tried again in March with new analyses of previous research, a gambit that doesn’t always fly with regulators.

An FDA advisory committee backed the drug in June, even though agency reviewers raised questions about its effectiveness and the potential for the treatment to cause inflammatory flare-ups. The final approval granted Wednesday came with a priority review voucher, which Ipsen can sell or use to speed the review process for another of its own drugs. The vouchers can fetch around $100 million when sold.

Regulators in other parts of the world have had mixed views of the treatment. Canada approved the medicine in early 2022 and it also has conditional approval in the United Arab Emirates. But the European Commission last month decided against authorization.

Even though FOP is exceedingly rare – affecting only about 900 people around the world – the condition has also drawn the interest of Regeneron Pharmaceuticals, which is testing a potential competing therapy in a Phase 3 trial. Ipsen also has a different option in Phase 2 that was acquired through the Clementia purchase.