Regeneron Pasatru, Genglycos Win FDA Approval
Get the Health newsletter
Daily health & science — research, biotech, public health, the studies worth knowing. Free.
- Regeneron Pharmaceuticals received FDA approval for Pasatru to treat fibrodysplasia ossificans progressiva after a three-decade development effort.
- Pasatru reduced new lesions that form bone by 94% in a Phase 3 trial, while the high dose produced a 90% reduction.
- Ultragenyx Pharmaceutical received FDA approval for Genglycos, which the report describes as the first gene therapy for a rare metabolic disorder, for adults and children with glycogen storage disease type Ia.
- Genglycos has a U.S. list price of $2.7 million per patient and is expected to be available through qualified treatment centers within 30 to 60 days.
Why it matters: For people with FOP, Pasatru’s Phase 3 trial reduced new lesion formation by 94%, with a 90% reduction at the high dose, offering a concrete treatment advance after a three-decade development effort. For the 1,500 to 2,500 U.S. patients with GSDIa, Genglycos carries a $2.7 million per-patient list price and access through qualified treatment centers within 30 to 60 days.
Ask SkimNews
