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US FDA’s Genetic Metabolic Diseases Adcomm Debuts With Zevra’s Arimoclomol

 
• By Sue Sutter

The new panel’s inaugural 2 August meeting will take up a challenging drug application, currently in its second review cycle, for Niemann-Pick disease type C, an ultra-rare condition without approved therapeutics.

Stage
The Genetic Metabolic Diseases Adcomm is scheduled for its first meeting on 2 August, although the committee's standing members have not yet been announced. • Source: Shutterstock

The US Food and Drug Administration’s Genetic Metabolic Diseases Advisory Committee (GeMDAC) will meet for the first time on 2 August to consider a therapeutic agent for a lysosomal storage disorder, a rare condition that the new panel seems tailor-made to handle.

Key Takeaways

  • The new Genetic Metabolic Diseases Advisory Committee will hold its first meeting on 2 August, although the FDA has yet to announce the committee's standing members.

  • The panel will consider Zevra’s arimoclomol for use in Niemann-Pick disease type C, an ultra-rare lysosomal storage disorder that has been the focus of recent meetings between the patient community and the FDA

The committee will

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