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FDA OKs Rare Bone Disorder Drug
20 Aug
Summary
- Regeneron's new drug treats fibrodysplasia ossificans progressiva.
- The drug significantly reduced new bone abnormalities.
- The rare disorder affects about 1 in 2 million people globally.
The U.S. Food and Drug Administration (FDA) has granted approval for Regeneron's experimental drug, garetosmab, marketed as Pasatru. This medication is intended for adult patients diagnosed with fibrodysplasia ossificans progressiva (FOP), a rare genetic disorder.
FOP is characterized by the gradual transformation of muscle, tendon, and ligament tissue into bone. This process creates what is often described as a "second skeleton," progressively limiting mobility and reducing life expectancy.
Clinical trials demonstrated garetosmab's efficacy, significantly reducing the development of new bone abnormalities. Patients receiving a 3 mg per kg dose experienced a 94% reduction, while those on a 10 mg per kg dose saw a 90% reduction compared to a placebo group.
This condition affects an estimated 1 in 2 million individuals globally, with around 800 to 900 diagnosed cases currently active worldwide, according to data from the National Institutes of Health.
