Regeneron’s ultra-rare bone disorder drug receives U.S. approval after 30-year development

Regeneron Pharmaceuticals announced that its therapy for an ultra-rare condition in which bone forms in soft tissue has been approved by the U.S. Food and Drug Administration.

The disease, sometimes called heterotopic ossification, affects a very small patient population and has no approved treatments. Regeneron’s drug, developed over three decades, is the first therapy to target the underlying cause.

The approval follows extensive clinical testing and a regulatory review that concluded the benefits outweigh the risks. The company expects the product to become commercially available later this year.

Separately, a U.S. appeals court partially revived a whistleblower lawsuit alleging that Regeneron made improper payments to physicians, but the court’s decision does not affect the newly granted approval.