FDA approves gene therapy for rare metabolic disease, Regeneron drug for bone disorder
The U.S. Food and Drug Administration approved Ultragenyx Pharmaceutical's gene therapy for glycogen storage disease type Ia, according to Reuters. The drug, branded as Genglycos, is the first approved gene therapy for the rare metabolic disorder in adults and children. GSDIa affects 1,500 to 2,500 patients in the U.S. and reduces the liver's ability to control glucose levels, leading to potentially life-threatening low blood sugar. The therapy will have a list price of $2.7 million per patient. Separately, STAT reported that Regeneron Pharmaceuticals won approval for Pasatru, a medicine for fibrodysplasia ossificans progressiva that caused a 94 percent reduction in new bone-forming lesions in a Phase 3 trial. The approval caps a three-decade effort. Patients with FOP, an ultra-rare disease that causes bone to grow where it should not, typically rely on wheelchairs by age 25 as bone accumulation locks up their limbs.
The approvals offer new treatment options for patients with rare inherited disorders that severely impair metabolic function and physical mobility. For FOP patients, the drug may help maintain mobility and extend lifespan beyond the typical 50s.
Written by the Genomes desk from the primary source linked above and checked against it. Research use only; not medical advice. Corrections: [email protected].