MIPLYFFA® (arimoclomol)
Niemann-Pick disease type C (NPC)
Filed (EU)Marketing Authorization Application submitted to EMA
Key Facts
Indication
Niemann-Pick disease type C (NPC)
Phase
Filed (EU)
Status
Marketing Authorization Application submitted to EMA
Company
About Zevra Therapeutics
Zevra Therapeutics is a purpose-driven rare disease company with a dual focus on commercial execution and late-stage clinical development. Its strategy involves following scientific data to build compelling regulatory narratives that address significant unmet patient needs. With a seasoned leadership team and a pipeline anchored in rare neurological and genetic disorders, Zevra aims to become a leader in the orphan drug space by advancing transformative therapies.
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