STAT+: FDA approves first treatment for ultra-rare bone disease

U.S. regulators on Wednesday approved the first treatment for an ultra-rare disease that causes people to grow bone where it otherwise shouldn’t be, extending an option to patients who have been advocating for access to the medicine.

The treatment, an oral medication that will be marketed as Sohonos, faced some questions about the quality of its data, and was rejected earlier this year by European regulators. But in approving the drug for the treatment of fibrodysplasia ossificans progressiva, or FOP, the Food and Drug Administration concluded that Sohonos’ benefits outweigh its risks.

The company behind Sohonos, the French drugmaker Ipsen, did not immediately disclose its price. Company executives have previously said the drug would be expensive, in line with treatments for other extremely rare diseases.

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