Headquartered in Boston, MA Fiscal year ends December NASDAQ: ZVRA zevra.com
Zevra Therapeutics is a commercial-stage rare disease biopharmaceutical company with a late-stage pipeline.
Its lead product is MIPLYFFA (arimoclomol), approved by the FDA in September 2024. It is used with miglustat to treat neurological manifestations of Niemann-Pick disease type C (NPC) in patients aged 2 and older, and it accounts for most of the company's revenue. Zevra is seeking to expand MIPLYFFA to infants under 24 months, with an sNDA planned for Q1 2027. It is also pursuing EU approval through a requested re-examination of its Marketing Authorisation Application. Arimoclomol is also supplied through a global Expanded Access Program, which generates net reimbursements.
Zevra also markets OLPRUVA (sodium phenylbutyrate) for certain urea cycle disorders.
Its main pipeline candidate is celiprolol for vascular Ehlers-Danlos syndrome. Celiprolol is in the Phase 3 DiSCOVER trial under an FDA Special Protocol Assessment and holds Orphan Drug and Breakthrough Therapy designations.
The company previously sold future royalties, intellectual property and other assets, and it has eliminated its long-term debt. Updated
A $610 million rare disease company just posted its fourth straight earnings beat, with MIPLYFFA net revenue of $34.0 million driving net income to $19.2 million. Next catalysts: a pediatric label expansion filing in Q1 2027 and the EU re-examination for arimoclomol.
Zevra Therapeutics is a commercial-stage rare disease biopharmaceutical company with a late-stage pipeline. Its lead product is MIPLYFFA (arimoclomol), approved by the FDA in September 2024.
The Wall Street consensus 12-month price target for ZVRA is $25.25.