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IntraBio’s Aqneursa Becomes Second Approved NPC Therapy In Four Days

 
• By Joseph Haas

IntraBio and Zevra will go head-to-head in the treatment of Niemann-Pick disease type C, with US FDA approvals of Aqneursa and Miplyffa, respectively, less than a week apart.

Niemann-Pick type C
IntraBio's Aqneursa becomes the second approved therapy for Niemann-Pick disease type C • Source: Shutterstock

The rare lysosomal storage disorder Niemann-Pick disease type C (NPC) now has two approved therapies as the US Food and Drug Administration gave a thumbs-up to IntraBio Inc.’s modified amino acid Aqneursa (levacetylleucine) on 24 September, just four days after the agency approved Zevra Therapeutics’ Miplyffa (arimoclomol) for the same disease.

IntraBio’s president of product development Taylor Fields noted that Aqneursa has been approved as a standalone therapy for NPC, while Mipflyffa’s indication specifies combination therapy with miglustat ( Johnson & Johnson’s Zavesca and generics), approved to treat

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