Zevra Therapeutics
Commercializes U.S.-approved therapies for Niemann-Pick type C disease and certain urea-cycle disorders.
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Profile
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
Key statistics
Year to date +38.6% · Average volume (30d) 1.2M
Analysts
Ratings
Target range
Latest analyst notes
| Date | Firm | Action | Rating | Prior target | Target |
|---|---|---|---|---|---|
| Jul 27, 2026 | Guggenheim | Reiterate | Buy | $29 | $24 |
| Jul 27, 2026 | BTIG | Reiterate | Buy | $24 | |
| Jul 27, 2026 | Canaccord Genuity | Reiterate | Buy | $29 | $20 |
| Jun 10, 2026 | Maxim Group | Reiterate | Buy | $25 | $35 |
| Jun 10, 2026 | Guggenheim | Reiterate | Buy | $25 | $29 |
Earnings
EPS
Revenue
History
| Report date | EPS est | EPS | Surprise | Revenue est | Revenue | Surprise | Next session |
|---|---|---|---|---|---|---|---|
| Aug 5, 2026Q2 FY26 | $0.11 | $0.14 | +27.3% | $33.9M | $39.7M | +17.1% | +12.82% |
| May 6, 2026Q1 FY26 | $0.06 | $0.18 | +200.0% | $32.0M | $36.2M | +13.3% | −2.22% |
| Mar 9, 2026Q4 FY25 | $0.05 | $1.16 | +2,201.6% | $28.1M | $34.1M | +21.6% | +21.27% |
| Nov 5, 2025Q3 FY25 | −$0.02 | −$0.01 | +50.0% | $26.6M | $26.1M | −2.2% | −9.73% |
Financial highlights
| 12 quarters | Q1 FY25 | Q2 FY25 | Q3 FY25 | Q4 FY25 | Q1 FY26 | Q2 FY26 | |
|---|---|---|---|---|---|---|---|
| Revenue | $20.4M | $25.9M | $26.1M | $34.1M | $36.2M | $39.7M | |
| Gross profit | $19.1M | $13.5M | $24.5M | $32.6M | $34.0M | $38.1M | |
| Operating income | −$5.4M | −$71.0M | $4.1M | $9.3M | $8.8M | $16.8M | |
| Net income | −$3.1M | $74.7M | −$544.0K | $12.2M | $37.9M | $8.8M | |
| Diluted EPS | −$0.06 | $1.36 | −$0.01 | $0.20 | $0.60 | $0.14 | |
| Gross margin | 93.4% | 52.2% | 94.0% | 95.5% | 93.7% | 96.2% | |
| Operating margin | -26.3% | -274.5% | 15.9% | 27.3% | 24.4% | 42.3% |
Fiscal years (4-quarter sums)FY21 $28.7M · FY22 $10.5M · FY23 $27.8M · FY24 $23.6M · FY25 $106.5M
Balance sheet, Jun 30, 2026Cash & short-term investments $221.6M · Total debt $1.5M · Total assets $302.7M · Shareholders' equity $217.7M
Dividends
No dividends on record.
Ownership
Shares outstanding 59.12M · Float 54.93M · 92.9% free float
Top shareholders13F · 13D/G · Form 4
| Holder | Shares | % outstanding | Form | Filed |
|---|---|---|---|---|
| BlackRock, Inc. | 5.11M | 8.64% | 13F | Aug 7, 2026 |
| FMR LLC | 4.92M | 8.32% | 13F | Aug 13, 2026 |
| Woodline Partners LP | 4.79M | 8.10% | 13F | Aug 14, 2026 |
| Vanguard Capital Management | 3.02M | 5.11% | 13G | Jul 31, 2026 |
| Nantahala Capital Management, LLC | 1.80M | 3.04% | 13F | Aug 19, 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 |
Peers
- Amgen
Branded nitrogen binder for chronic UCD management
- Rafael
Phase 3 NPC1 therapy approaching a potential U.S. filing
- Jazz Pharmaceuticals
Only FDA-approved adult idiopathic-hypersomnia therapy
- Alkermes
Two IH programs spanning oxybate and orexin mechanisms
CompareAfter-hours close
| Company | Price | Change | |||||
|---|---|---|---|---|---|---|---|
| Zevra Therapeutics | $11.57 | +0.04% | $683.7M | 12.4 | 13.8 | 5.0 | +53.3% |
| Amgen | $430.27 | −0.49% | $233.38B | 26.9 | 18.4 | 6.1 | +9.7% |
| Rafael | $2.59 | +2.37% | $131.1M | — | — | 133.8 | −50.6% |
| Jazz Pharmaceuticals | $244.54 | +0.02% | $15.36B | 16.7 | 9.7 | 3.3 | +15.6% |
| Alkermes | $47.25 | −0.34% | $7.95B | 123.8 | — | 4.8 | +27.0% |
| Median | 26.9 | 14.1 | 5.4 | +12.6% |






