- BridgeBio Pharma ( NASDAQ: BBIO ) and Sentynl Therapeutics' said a committee of the European Medicines Agency (EMA) recommended approval under exceptional circumstances of their therapy Nulibry to treat patients with molybdenum cofactor deficiency (MoCD) Type A.
- MoCD is a rare inherited metabolic disorder characterized by brain dysfunction (encephalopathy) and intractable seizures seen in certain babies a few days after birth.
- Nulibry (fosdenopterin) was approved in the U.S. in February 2021 for the same use.
- The European Commission (EC) will now make a final decision, which generally follows the recommendation made by the EMA's Committee for Medicinal Products for Human Use (CHMP), whether to approve the drug.
- If approved by the EC, NULIBRY would be the first and only approved therapy in the EU to treat patients with MoCD type A, the company said in a July 25 press release.
- BridgeBio said the CHMP decision was backed by data from three clinical trials.
- In March, BridgeBio ( BBIO ) and Sentynl, a unit of Zydus Lifesciences, signed an agreement over Nulibry.
- BBIO +2.36% to $10.39 premarket July 25
For further details see:
BridgeBio, Sentynl's Nulibry get EMA panel nod for approval in EU