• The FDA has approved Zevra Therapeutics' Miplyffa for the treatment of Niemann-Pick disease type C, a rare neurodegenerative disorder.
• This regulatory milestone marks a significant advancement in the treatment landscape for this progressive disease, offering new hope to patients.
• Following the announcement, Zevra Therapeutics Inc. (ZVRA) experienced a surge in its stock price, reaching a new 52-week high.
• The approval underscores the potential of Miplyffa to address the unmet medical needs of individuals affected by Niemann-Pick disease type C.