BridgeBio : FDA Approves NULIBRY To Reduce Risk In Neurological Disease

 BridgeBio Pharma Inc. (BBIO) and affiliate Origin Biosciences Inc. said that the U.S. Food and Drug Administration has approved NULIBRY or fosdenopterin for Injection as the first therapy to reduce the risk of mortality in patients with molybdenum cofactor deficiency or MoCD Type A.

Molybdenum cofactor deficiency Type A is an ultra-rare and progressive condition, known to impact less than 150 patients globally with a median survival of four years. MoCD Type A presents shortly after birth, often with severe encephalopathy and intractable seizures. NULIBRY is a first-in-class approved cPMP substrate replacement therapy.

MoCD Type A is an autosomal recessive, inborn error of metabolism caused by mutations in the molybdenum cofactor synthesis 1 gene and characterized by a deficiency in molybdenum cofactor production, leading to a lack of molybdenum-dependent enzyme activity. The lack of activity leads to decreased sulfite oxidase activity with buildup of sulfite and secondary metabolites in the brain, which causes irreversible neurological damage.

https://www.nasdaq.com/articles/bridgebio-%3A-fda-approves-nulibry-to-reduce-risk-of-mortality-in-patients-with-mocd-type-a