FDA approves Regeneron's Pasatru for rare bone disorder
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The U.S. FDA on Wednesday approved Regeneron's drug Pasatru for adults with fibrodysplasia ossificans progressiva, a rare genetic disorder that turns soft tissue into bone. Regeneron shares rose 4% after the approval. The therapy reduced new bone abnormalities by 94% at a 3 mg/kg dose in a 56-week trial of 63 participants.
Key Facts
- FDA approved Regeneron's Pasatru for adults with fibrodysplasia ossificans progressiva.
- In a 56-week trial of 63 participants, Pasatru reduced new bone abnormalities by 94% at a 3 mg/kg dose and 90% at a 10 mg/kg dose versus placebo.
- Regeneron shares rose 4% after the approval.
- Ipsen's Sohonos, approved in 2023, is the only other FDA-approved treatment for the condition.
- The condition affects roughly 1 in 2 million people worldwide, with 800-900 active diagnosed cases globally.
FDA Approval of Pasatru
The U.S. FDA on Wednesday approved Regeneron's garetosmab, branded as Pasatru, for treating adults with fibrodysplasia ossificans progressiva. The disease causes muscle, tendon and ligament tissue to gradually turn into bone, leading to a "second skeleton" and progressive loss of mobility. In a 56-week trial involving 63 participants, Pasatru reduced the development of new bone abnormalities by 94% in patients treated with a 3 mg per kg dose and by 90% at a 10 mg per kg dose compared with placebo. Pasatru works by blocking Activin A, a protein involved in triggering abnormal bone growth in patients with FOP.
Market and Competitors
New York-based Regeneron's shares rose 4% after the approval. The drug will compete with French drugmaker Ipsen's oral treatment Sohonos, which in 2023 became the only other treatment approved by the U.S. Food and Drug Administration. Incyte and partner Mirum Pharma, as well as privately held Ashibio, are also developing treatments for the condition. In 2020, Regeneron paused dosing in a mid-stage trial of the drug after five patient deaths, ultimately discontinuing the study and working with global regulators to design the late-stage trial.
Disease Burden
The condition affects roughly 1 in 2 million people worldwide. Around 800 to 900 active diagnosed cases exist globally, according to National Institutes of Health data. Regeneron plans to start a trial for children later this year, according to clinical team member Susan Rhee.
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FDA approves Regeneron's Pasatru for rare bone disorder
