BridgeBio : FDA Approves NULIBRY To Reduce Risk Of Mortality In Patients With MoCD Type A

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.

For comments and feedback contact: editorial@rttnews.com

Business News

Editors Pick
Shares of Apple Inc. (AAPL) jumped nearly 5% in extended trading session on Thursday after the California-based tech giant reported first-quarter results, with both earnings and revenues trumping Wall Street's expectations, driven by strong demand for its latest iPhone 13 and other services. Apple... The U.S. Food and Drug Administration has revised the authorizations for two monoclonal antibody treatments - bamlanivimab and etesevimab (administered together) and REGEN-COV (casirivimab and imdevimab) - to limit their use to only when the patient is likely to have been infected with or exposed to a variant that is susceptible to these treatments. Comcast Corp. (CMCSA, CMCSK) on Thursday reported that net income attributable to Comcast for the quarter decreased 9.6 percent to $3.06 billion or $0.66 per share from $3.38 billion or $0.73 per share in the year-ago quarter. Excluding items, adjusted earnings for the quarter were $0.77, compared to...
Follow RTT