Zevra Therapeutics Inc. will try to follow in the footsteps of Acadia Pharmaceuticals Inc. with Rett syndrome drug Daybu (trofinetide) by asking the EMA’s Committee for Medicinal Products for Human Use (CHMP) to rethink arimoclomol for Niemann-Pick disease type C (NPC). Boston-based Zevra will request a second look at the MAA after CHMP gatekeepers ruled that efficacy in NPC has not been sufficiently proven.

Niagen Bioscience Inc.’s proprietary lead small-molecule drug candidate, NB-4168, has been awarded European orphan drug designation and U.S. rare pediatric disease designation for the treatment of ataxia telangiectasia.

The EMA is recommending withdrawing Amgen Inc.’s complement inhibitor Tavneos (avacopan) from the market in Europe, saying data provided at the time it assessed the MAA “were found to be incorrect and misleading and could no longer be relied upon for demonstrating Tavneos’ effectiveness.”