STAT+: Pharmalittle: We’re reading about a Regeneron rare disease drug, an Ultragenyx gene therapy, and more

Regeneron's Pasatru, a medicine for the ultra-rare disease fibrodysplasia ossificans progressiva (FOP), has won U.S. regulatory approval after a three-decade effort. FOP causes bone to form in muscles, tendons, and other soft tissues, locking joints; many patients rely on wheelchairs by age 25 and only some live into their 50s. In a Phase 3 trial, the drug produced a 94% reduction in new bone-forming lesions, with a high dose achieving a 90% reduction. The hope is that Pasatru will help patients maintain mobility and potentially extend survival.

The FDA also approved Ultragenyx's Genglycos, the first gene therapy for glycogen storage disease type Ia (GSDIa, also known as Von Gierke disease), in adults and children. The condition, which affects an estimated 1,500–2,500 patients in the U.S., impairs the liver's ability to control glucose levels, leading to potentially life-threatening low blood sugar. Genglycos carries a U.S. list price of $2.7 million per patient and will be available through qualified treatment centers within 30–60 days.