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Zevra’s Miplyffa Wins First-Ever US Approval, But In Combination With Off-Label Miglustat

Drug’s List Price Is $1m Per Year On Average

 
• By Mandy Jackson

Miplyffa (arimoclomol) was approved for Niemann-Pick disease type C when administered with another high-priced drug used off label as standard of care for about 80% of NPC patients. 

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Zevra will pull the lever on its Miplyffa launch in eight to 12 weeks • Source: Shutterstock

Zevra Therapeutics Inc. received the first-ever US Food and Drug Administration approval in Niemann-Pick disease type C (NPC) for Miplyffa (arimoclomol), but faces some complicating factors: co-administration with an off-label drug, a hefty combined price tag, and a competitor that could be approved imminently.

Miplyffa was cleared on 20 September to treat neurological symptoms associated with NPC in children and adults aged 2 and...

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