ZEVRA THERAPEUTICS, INC.
ZVRA evidence brief
What we can confirm
- BioSniper Score:
- 7.8 / 10
- Market Cap:
- $716.2M
- Lead Asset:
- apadaz (Approved)
What it means
What to watch
Prepared research question
What matters most for ZVRA—catalysts, financial risk and supporting evidence?
Traceable citations · Unknowns marked
BioSniper AI · ZVRA
Zevra Therapeutics is a rare-disease company focused on commercializing MIPLYFFA (arimoclomol) for Niemann-Pick disease type C and advancing a late-stage pipeline including celiprolol.
Key risk: Single-product revenue concentration
BioSniper Score
Based on 5 of 6 signals
Financial data as of Jun 30, 2026.
Tracking 6 pipeline candidates · 1 clinical trial · 4 catalysts · 1 FDA decision for ZEVRA THERAPEUTICS, INC..
Zevra Therapeutics is a rare-disease company focused on commercializing MIPLYFFA (arimoclomol) for Niemann-Pick disease type C and advancing a late-stage pipeline including celiprolol.
- Lead product MIPLYFFA (arimoclomol) received FDA approval on Sep. 20, 2024 for Niemann-Pick disease type C (NPC) [sec_mda · as of 2026-08-05].
- Full-year 2025 net revenue was $106.5 million, including $87.4 million from MIPLYFFA [sec · filed 2026-04-20].
- As of 2026-06-30, the company reported cash & equivalents of $145.3M, total revenue of $39.7M, net income of $8.8M, and shareholders' equity of $217.7M [sec].
- An EU Marketing Authorization Application (MAA) for arimoclomol was submitted in July 2025 [sec · filed 2026-04-20].
- The Phase 3 DiSCOVER trial of celiprolol had approximately one-third of patients enrolled as of year-end 2025 [sec · filed 2026-04-20].
What works
- MIPLYFFA is generating substantial revenue ($87.4M in 2025) and has orphan drug exclusivity through 2031, providing a stable commercial foundation [sec · filed 2026-04-20] [sec_mda · as of 2026-08-05].
- The company has a strong cash position ($145.3M as of 2026-06-30) and positive net income ($8.8M), indicating financial stability [sec].
- Global expansion is underway with an EU MAA submitted and an Expanded Access Program serving 113 patients, broadening market reach [sec · filed 2026-04-20].
- The company is advancing a late-stage pipeline (celiprolol Phase 3) and has a disciplined strategy focused on rare diseases [sec · filed 2026-04-20].
What to weigh
- Revenue is heavily concentrated on a single product (MIPLYFFA), creating dependency risk [sec · filed 2026-04-20].
- The company discontinued in-house drug discovery and closed lab facilities in 2024, reducing internal R&D capacity [sec_mda · as of 2025-11-05].
- Celiprolol is still in Phase 3 with only one-third enrolled as of year-end 2025, so its commercial contribution is years away [sec · filed 2026-04-20].
- Patent expirations for several products (e.g., acetaminophen/benzhydrocodone in 2031) could affect future IP exclusivity [upcoming catalysts].
From the filings
Quoted directly from source documents.
MIPLYFFA FDA approval date and orphan drug exclusivity
“MIPLYFFA | FDA Approval: Sep. 20, 2024; Orphan Drug Exclusivity ("ODE") through 2031”
SEC MD&A · August 5, 2026
2025 net revenue and MIPLYFFA contribution
“For the full year, Zevra generated $106.5 million in net revenue, including $87.4 million from MIPLYFFA.”
SEC filings · April 20, 2026
EU MAA submission for arimoclomol
“We submitted the EU Marketing Authorization Application (MAA) for arimoclomol in July of 2025”
SEC filings · April 20, 2026
Celiprolol Phase 3 enrollment status
“we advanced our Phase 3 DiSCOVER trial of celiprolol, our late-stage development asset, with approximately one-third of patients enrolled as of year‑end 2025.”
SEC filings · April 20, 2026
Catalyst timeline
- 2031-02-22 · Patent_Expiry · ACETAMINOPHEN; BENZHYDROCODONE HYDROCHLORIDE — Patent expiry for NDA208653; may allow generic competition for these products.
- 2031-09-20 · Patent_Expiry · ARIMOCLOMOL CITRATE — Patent expiry for NDA214927; could affect exclusivity of arimoclomol citrate formulations.
- 2036-10-17 · Patent_Expiry · SODIUM PHENYLBUTYRATE — Patent expiry for NDA214860; long-term IP milestone for this product.
- 2037-12-09 · Patent_Expiry · DEXMETHYLPHENIDATE HYDROCHLORIDE; SERDEXMETHYLPHENIDATE CHLORIDE — Patent expiry for NDA212994; potential entry of generics for these stimulant products.
Risks & what to watch (4)›
Key risks
- Single-product revenue concentration — MIPLYFFA contributed 82% of 2025 net revenue, leaving the company vulnerable to any disruption in that product's sales [sec · filed 2026-04-20].
- Pipeline dependency on celiprolol — Celiprolol is the only late-stage development asset, and its Phase 3 trial is only one-third enrolled, with no near-term approval expected [sec · filed 2026-04-20].
- Patent expirations on legacy products — Patents for acetaminophen/benzhydrocodone and arimoclomol citrate expire in 2031, potentially opening competition [upcoming catalysts].
- Discontinued internal R&D — In-house drug discovery and lab facilities were closed in 2024, limiting the company's ability to generate new pipeline candidates internally [sec_mda · as of 2025-11-05].
What to watch
- EU regulatory decision on the arimoclomol MAA, which could open a major new market [sec · filed 2026-04-20].
- Enrollment progress and eventual data from the Phase 3 DiSCOVER trial of celiprolol [sec · filed 2026-04-20].
- Global expansion execution, including new distribution agreements and EAP patient numbers [sec · filed 2026-04-20].
- Patent expirations in 2031 for key products, which may affect long-term revenue exclusivity [upcoming catalysts].
Sources: SEC filings · SEC MD&A · Generated October 12, 2026 · How we verify sources →
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Evidence & data
Score breakdownBased on 5 of 6 signals · derived from public data
Cash runway data unavailable
D/E 0.39 · 4% dilution
Lead: Approved
6 candidates
4 upcoming, next in ~1592d
0 buys · 4 sells (180d)
Catalysts
Pipeline
Regulatory
Financials
Institutional Holdings
Source: SEC Form 13F · as of Mar 2026Institutional holders
148
Total value held
$381.6M
| Holder | Shares | Value |
|---|---|---|
| blackrock, inc. | 4,456,476 | $41.5M |
| woodline partners lp | 3,450,943 | $32.2M |
| fmr llc | 3,211,877 | $29.9M |
| nantahala capital management, llc | 2,521,936 | $23.5M |
| vanguard capital management llc | 2,469,328 | $23.0M |
| state street corp | 1,561,569 | $14.6M |
| geode capital management, llc | 1,371,857 | $12.8M |
| aigh capital management llc | 1,200,000 | $11.2M |
| morgan stanley | 1,192,262 | $11.1M |
| legal & general group plc | 1,163,090 | $10.8M |
Showing top 10 of 148 institutional holders of ZEVRA THERAPEUTICS, INC.. Open the full 13F history after sign-in.
Short Interest
as of Apr 28, 2026- % of float
- 8.2%
- Days to cover
- 5.9
- Shares short
- 4,776,072
- Daily short volume
- -
Short percent of float and days-to-cover reflect reported short positioning. Daily short volume is the exchange-reported short share of a single day's trading volume and is noisier. See the most-shorted biotech ranking →
ZVRA short interestGovernance
Frequently asked questions
What is the AI investment thesis for ZEVRA THERAPEUTICS, INC.?
Zevra Therapeutics is a rare-disease company focused on commercializing MIPLYFFA (arimoclomol) for Niemann-Pick disease type C and advancing a late-stage pipeline including celiprolol.
What is ZEVRA THERAPEUTICS, INC.'s lead drug candidate?
ZEVRA THERAPEUTICS, INC.'s most advanced tracked candidate is apadaz (Approved).
What is ZEVRA THERAPEUTICS, INC.'s next catalyst?
ZEVRA THERAPEUTICS, INC.'s next tracked catalyst is a Patent_Expiry event — "Patent_Expiry: NDA208653" — expected February 22, 2031.
Who is the CEO of ZEVRA THERAPEUTICS, INC.?
Mr. Neil F. McFarlane is the chief executive officer of ZEVRA THERAPEUTICS, INC. (ZVRA).
Where is ZEVRA THERAPEUTICS, INC. headquartered?
ZEVRA THERAPEUTICS, INC. (ZVRA) is headquartered in Boston, MA, United States.
The AI overview and BioSniper Score are neutral, data-derived signals built from public SEC filings, ClinicalTrials.gov and FDA records — not investment advice.