Zevra Therapeutics (ZVRA)
Commercializes U.S.-approved therapies for Niemann-Pick type C disease and certain urea-cycle disorders.
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Commercializes MIPLYFFA for neurological manifestations of Niemann-Pick disease type C and OLPRUVA for certain urea-cycle-disorder patients. The U.S. rare-disease portfolio grew from the arimoclomol asset purchase and Acer acquisition.
Commercializes MIPLYFFA, an arimoclomol therapy approved in the United States for neurological manifestations of Niemann-Pick disease type C in adults and pediatric patients when used with miglustat. It also sells OLPRUVA for certain people with urea-cycle disorders. Together, these U.S.-approved therapies make it a commercial-stage rare-disease company.
The current portfolio stems from the acquisition of arimoclomol assets from Orphazyme and the later purchase of Acer Therapeutics. Commercial operations are centered on U.S.-approved therapies, while the former SDX portfolio has been divested. The business is shaped by product-specific FDA approvals; MIPLYFFA’s approved use for that condition remains in combination with miglustat.
Company facts
Target rangeAs of Sep 22, 2026
Latest analyst notes
| Date | Firm | Action | Prior | Current |
|---|---|---|---|---|
| Jul 27, 2026 | Guggenheim | Reiterate | Rating: —Target: $29 | Target: $24 |
| Jul 27, 2026 | BTIG | Reiterate | Rating: —Target: — | Target: $24 |
| Jul 27, 2026 | Canaccord Genuity | Reiterate | Rating: —Target: $29 | Target: $20 |
| Jun 10, 2026 | Maxim Group | Reiterate | Rating: —Target: $25 | Target: $35 |
| Jun 10, 2026 | Guggenheim | Reiterate | Rating: —Target: $25 | Target: $29 |
EPSReportedEstimate
RevenueReportedEstimate
Balance sheet, Jun 30, 2026Cash & short-term investments $221.6M · Total debt $1.5M · Total assets $302.7M · Shareholders' equity $217.7M
- Revenue
- $39.7M
- Operating income
- $16.8M
- Net income
- $8.8M
No dividends on record.
| Institutions | ≈ 75.60% | 44.90M shares | |
|---|---|---|---|
| 168 holders · as of Jun 30, 2026 · largest filers plus last quarter's tail | |||
| Insiders | 1.38% | 819.0K shares | |
| 10 holders · as of Aug 17, 2026 | |||
| Other | ≈ 23.02% | 13.67M shares | |
| remainder | |||
59.38M company shares as of Sep 23, 2026 · institutions as of Jun 30, 2026 · holders as of their latest filing
This quarter's institutional total failed a plausibility check; shown: the largest filers plus last quarter's remainder.
Top shareholders13F · 13D/G · Form 4
| Holder | Stake | Shares | Date |
|---|---|---|---|
BlackRock, Inc.Institution | 8.60% | 5.11M | as of Jun 30, 2026 |
FMR LLCInstitution | 8.28% | 4.92M | as of Jun 30, 2026 |
Woodline Partners LPInstitution | 8.06% | 4.79M | as of Jun 30, 2026 |
Vanguard Capital Management LLCInstitution | 4.44% | 2.64M | as of Jun 30, 2026 |
Nantahala Capital Management, LLCInstitution | 3.03% | 1.80M | as of Jun 30, 2026 |
Insider tradesForm 4
| Date | Insider | Role | Shares | Avg price |
|---|---|---|---|---|
| Aug 14, 2026 | Rahsaan Thompson | officer: Chief Legal & Compliance | −42,666 | $11.13 |
| Aug 10, 2026 | Corey Michael Watton | director | −12,300 | $11.25 |
| Jun 22, 2026 | Rahsaan Thompson | officer: Chief Legal & Compliance | −34,557 | $12.68 |
| May 11, 2026 | Rahsaan Thompson | officer: Chief Legal & Compliance | −1,794 | $11.30 |
| Feb 9, 2026 | Neil F. McFarlane | director, officer: President and CEO | −14,625 | $8.56 |
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Compare
| Company | Price | Change | |||||
|---|---|---|---|---|---|---|---|
| Zevra Therapeutics | $11.29 | −2.50% | $684.6M | 12.4 | 13.5 | 5.0 | +53.3% |
| Amgen | $408.49 | −0.43% | $221.41B | 25.5 | 17.4 | 5.8 | +9.7% |
| Rafael | $1.97 | −1.50% | $102.4M | — | — | 104.5 | −50.6% |
| Jazz Pharmaceuticals | $236.93 | −1.51% | $15.11B | 16.4 | 9.4 | 3.3 | +15.6% |
| Alkermes | $42.30 | −2.29% | $7.26B | 113.0 | — | 4.4 | +27.0% |
| Median | 25.5 | 13.4 | 5.1 | +12.6% |