Zevra Therapeutics Inc. (ZVRA), which emerged as a commercial-stage company in 2024, is continuing its transformation into a patient-focused leader in rare disease therapeutics. The company's shares touched a 52-week high of $10.03 last week.
The lead product of the company is MIPLYFFA (arimoclomol), an orally delivered treatment for Niemann-Pick disease type C, an ultra-rare, progressive, and neurodegenerative disorder. Approved by the U.S. regulatory agency in September 2024, the drug was commercially launched the following month. The company is expected to seek European Medicines Agency's approval for MIPLYFFA during the second half of this year.
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