Zevra receives negative EU opinion on arimoclomol
By Jon CuthbertKey points
- Zevra Therapeutics (NASDAQ:ZVRA) received a negative recommendation from the European Medicines Agency's CHMP following its review of arimoclomol for Niemann-Pick disease type C.
- The European Commission is expected to issue its final decision within 67 days, while Zevra plans to continue providing arimoclomol to eligible patients through its Expanded Access Program.
- The application was supported by Phase 2/3 NPC 002 results showing reduced disease progression over 12 months when arimoclomol was combined with miglustat.
Zevra Therapeutics (NASDAQ:ZVRA) announced that the European Medicines Agency's Committee for Medicinal Products for Human Use maintained a negative recommendation on arimoclomol following re-examination of its European marketing authorization application for Niemann-Pick disease type C.
The European Commission is expected to issue its legally binding decision within 67 days of the committee's opinion, leaving the medicine's European marketing authorization pending.
"We are deeply disappointed by the CHMP’s opinion, particularly given the significant unmet medical need faced by individuals and families living with NPC," said Neil F. McFarlane, President and Chief Executive Officer of Zevra Therapeutics.
Zevra will continue supporting eligible patients already receiving arimoclomol through its global Expanded Access Program, subject to regulatory requirements and coordination with national authorities.1
The application included results from the pivotal NPC 002 Phase 2/3 trial, which showed reduced disease progression over 12 months when arimoclomol was combined with miglustat, while long-term open-label extension data indicated sustained treatment effects over multiple years.
In its earlier assessment, the EMA questioned the reliability of the trial data analysis and found no demonstrated benefits in walking ability and cognition, although Zevra continues marketing MIPLYFFA in the United States for NPC.
