ZVRA combines fast-growing, high-margin MIPLYFFA revenue and a debt-free balance sheet with substantial regulatory and celiprolol execution upside.
Overview
Zevra Therapeutics (ZVRA) is a commercial-stage rare-disease biopharmaceutical company whose revenue base is increasingly driven by MIPLYFFA, the first FDA-approved therapy designed to target the underlying pathophysiology of Niemann-Pick disease type C, alongside the smaller OLPRUVA franchise and global expanded-access programs. MIPLYFFA benefits from orphan exclusivity through September 2031 and Orange Book protection through 2041, while high switching costs and AmplifyAssist support recurring, low-attrition revenue. **Q2 2026 revenue rose 53.3% year over year to $39.66 million**, including $30.20 million of MIPLYFFA U.S. sales and $9.00 million of global EAP revenue, while adjusted net income reached $15.20 million, or $0.25 per diluted share. The company also holds $260.2 million of cash and short-term investments and zero long-term debt after repaying its $63.1 million term loan. The key valuation debate is whether European access is restored and celiprolol succeeds in Phase 3. At a current price of $11.88, the report estimates a probability-weighted five-year value of $30.71, versus consensus analyst targets of approximately $25.11–$25.33. Near-term catalysts include the Q4 2026 European re-examination, FDA engagement on celiprolol, and continued U.S. patient enrollment.