BridgeBio Pharma’s, Origin Biosciences’ Nulibry Nabs FDA Approval for Fatal Rare Genetic Metabolism Disorder
The FDA has approved Nulibry (fosdenopterin) from BridgeBio Pharma and its affiliate company Origin Biosciences as an injection meant to treat Molybdenum Cofactor Deficiency Type A, a rare genetic metabolism disorder that causes seizures, brain injury and death.
According to the FDA, patients with the disorder suffer “severe and rapidly progressive neurologic damage, including intractable seizures, feeding difficulties and muscle weakness” from toxins accumulating in metabolic substances, with the only previous treatment options consisting of supportive care and drugs that address complications stemming from the disease. Most patients die very young in childhood from the disease.
Administered intravenously, Nulibry works by replacing a missing substance in patients called cyclic pyranopterin monophosphate. Efficacy data showed the drug was associated with a survival rate of 84 percent at three years for 13 treated patients vs. a survival rate of 55 percent at three years in 18 patients who were untreated.
Nulibry had secured Priority Review, Breakthrough Therapy and Orphan Drug designations from the FDA.