- The European Commission (EC) granted marketing authorization to BridgeBio Pharma ( NASDAQ: BBIO ) and Sentynl Therapeutics' Nulibry to treat patients with molybdenum cofactor deficiency (MoCD) Type A.
- Nulibry (fosdenopterin) for injection is the first therapy approved for this condition, — known to impact less than 150 patients globally with a median survival of four years — the company said in a Sept. 20 press release.
- MoCD is a rare inherited metabolic disorder characterized by brain dysfunction (encephalopathy) and intractable seizures seen in certain babies a few days after birth.
- In July, a panel of the European Medicines Agency (EMA) recommended the approval of Nulibry.
- The EC approval was backed by data from three clinical trials.
- Nulibry was approved in the U.S. in February 2021 for the same use.
- In March, BridgeBio ( BBIO ) and Sentynl, a unit of Zydus Lifesciences, signed an agreement over Nulibry.
For further details see:
BridgeBio, Sentynl's Nulibry for rare disorder in children gets approval in EU