• Miplyffa (arimoclomol), the first ever medication specifically approved for Niemann-Pick disease type C, is now available from Zevra Therapeutics (NASDAQ:ZVRA).
• The drug is approved for use in combination with Johnson & Johnson's (JNJ) Zavesca (miglustat) for the treatment of patients two years and older. Zavesca is approved for Gaucher disease.
• Miplyffa works by boosting the activation of the transcription factors EB (TFEB) and E3 (TFE3), leading to the upregulation of coordinated lysosomal expression and regulation (CLEAR) genes.
• The U.S. FDA approved Miplyffa in September.

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