Zevra Therapeutics
Zevra Therapeutics is a commercial-stage biopharmaceutical company developing and commercializing therapies for rare diseases, including the first FDA-approved treatment for Niemann-Pick disease type C (MIPLYFFA), an approved urea cycle disorder therapy (OLPRUVA), and a Phase 3 asset (celiprolol) for vascular Ehlers-Danlos syndrome.
- Company typePublic
- Founded2022
- HeadquartersOrlando, United States
- Headcount51–100
- GTM typeB2B
- OfferingHardware or Manufacturing
What Zevra Therapeutics does
Zevra Therapeutics, Inc. (NASDAQ: ZVRA) is a commercial-stage rare disease biopharmaceutical company headquartered in Boston, Massachusetts with additional operations in Celebration, Florida and Frederiksberg, Denmark. The company, formerly KemPharm, Inc., pivoted from its prodrug heritage to rare disease drug development in January 2022, was renamed Zevra Therapeutics on Rare Disease Day 2023, and built its current portfolio through two acquisitions: Orphazyme A/S (May 2022, arimoclomol) and Acer Therapeutics (November 2023, OLPRUVA and celiprolol). Its two FDA-approved commercial products are MIPLYFFA (arimoclomol), the first and only approved treatment for Niemann-Pick disease type C (NPC) in adult and pediatric patients aged 2 and older, used in combination with miglustat; and OLPRUVA (sodium phenylbutyrate), a dual-coated oral suspension for certain urea cycle disorders. A third program, celiprolol, is in Phase 3 development (DiSCOVER trial) for COL3A1-positive vascular Ehlers-Danlos syndrome (VEDS).
The company's underlying technology combines a proprietary Ligand Activated Therapy (LAT) prodrug platform — inherited from KemPharm — with rare disease drug development capabilities, including arimoclomol's novel heat shock protein amplification mechanism that addresses lysosomal dysfunction and celiprolol's selective adrenoceptor modulation that reduces mechanical stress on vascular walls. Real-world data generation through a multi-year U.S. and international Expanded Access Program (covering 122 and 113 patients respectively) is used to inform both clinical development strategy and payer engagement.
Zevra operates a specialty pharmaceutical commercial model: a direct specialty sales force targets healthcare professionals treating patients with NPC, UCDs, and VEDS; distribution in the U.S. runs through an exclusive specialty pharmacy arrangement with Orsini, while international distribution outside Europe is handled by Uniphar under a December 2025 exclusive agreement. Revenue is generated from commercial product sales (MIPLYFFA U.S. net sales of $24.6 million in Q1 2026), Expanded Access Program reimbursements ($10.2 million in Q1 2026), and one-time divestiture proceeds (the $50 million SDX portfolio sale to Commave Therapeutics in March 2026). FY2025 total net revenue reached $106.5 million (up 350% year-over-year), the company reported $83.2 million in net income, and as of Q1 2026 it held $236.8 million in cash with no outstanding debt after fully repaying a $63 million term loan.
Zevra Therapeutics firmographics
Firmographics- Name
- Zevra Therapeutics
- Legal name
- Zevra Therapeutics, Inc.
- Website
- https://zevra.com
- Company type
- Public
- Founded year
- 2022
- Operating status
- Operating
- Headcount range
- 51–100 employees
- Short description
- Zevra Therapeutics is a commercial-stage biopharmaceutical company developing and commercializing therapies for rare diseases, including the first FDA-approved treatment for Niemann-Pick disease type C (MIPLYFFA), an approved urea cycle disorder therapy (OLPRUVA), and a Phase 3 asset (celiprolol) for vascular Ehlers-Danlos syndrome.
- Ownership category
- akta.pro rank
Zevra Therapeutics industry classification
Industry- Product category
- Rare Disease Therapeutics
- NAICS
- Biological Product (except Diagnostic) Manufacturing (325414)
- SIC
- Pharmaceutical Preparations (2834)
- akta.pro primary industry
- Rare Neurology & Neurodegenerative Disorder Therapies (HLAIAIAD)
- akta.pro secondary industry
- Rare Gastroenterology & Hepatology Disorder Therapies (HLAIAIAL)
Keywords
Where Zevra Therapeutics is headquartered
LocationHeadquarters
- HQ city
- Orlando
- HQ country
- United States
- HQ region
- North America
Offices3 records
Markets served
Zevra Therapeutics business model
Business model- GTM type
- B2B
- Offering type
- Hardware or Manufacturing
- Cost components
- Personnel, Technology or R&D, Supply Chain, Marketing or Sales, Operations
Revenue model
- Commercial product sales: Direct sales of approved pharmaceutical products MIPLYFFA (arimoclomol) for NPC and OLPRUVA (sodium phenylbutyrate) for UCDs; Q1 2026 US MyPlifer net sales of $24.6 million
- Expanded Access Program (EAP) reimbursements: Reimbursements from expanded access programs covering 122 patients across multiple territories; $10.2 million in Q1 2026; 113 international patients enrolled
- Asset divestiture (one-time): Sale of SDX portfolio including AZSTARYS and KP1077 to Commave Therapeutics for $50 million in March 2026 as part of litigation settlement and balance sheet restructuring
- Royalty income (third-party): Royalties received from third parties (Xoma Royalty/Ligand) tied to Miplyffa commercial sales as part of historical royalty monetization arrangements
Pricing tiers
| Model | Billing | Price |
|---|---|---|
| Other | — | Specialty pharmacy pricing for MIPLYFFA via Orsini |
Go-to-market motion5 records
Distribution channels5 records
Marketing channels7 records
Zevra Therapeutics product offering
Product offeringCore offering
Zevra Therapeutics is a commercial-stage rare disease biopharmaceutical company that develops and commercializes therapies for ultra-rare diseases. Its portfolio includes two FDA-approved products: MIPLYFFA (arimoclomol), the first FDA-approved treatment for Niemann-Pick disease type C (NPC), and OLPRUVA (sodium phenylbutyrate) for urea cycle disorders (UCDs). The company also has a late-stage pipeline asset, celiprolol, in a Phase 3 trial for Vascular Ehlers-Danlos Syndrome (VEDS). Distribution is conducted via a specialty pharmacy (Orsini) in the US and an international distribution partner (Uniphar) outside Europe.
Product overview
Zevra Therapeutics operates a focused rare disease therapeutics portfolio rather than a unified software platform. The offering centers on two FDA-approved commercial products — MIPLYFFA® (arimoclomol) for Niemann-Pick disease type C and OLPRUVA® (sodium phenylbutyrate) for certain urea cycle disorders — complemented by a late-stage clinical asset, Celiprolol, currently in a Phase 3 DiSCOVER trial for Vascular Ehlers-Danlos Syndrome (VEDS). Arimoclomol is also progressing through a Marketing Authorization Application review with the European Medicines Agency to expand the NPC franchise internationally. The portfolio was assembled through two key acquisitions: Orphazyme A/S (May 2022, brought arimoclomol) and Acer Therapeutics (November 2023, brought OLPRUVA and celiprolol), positioning Zevra as a commercial-stage rare disease company executing on commercial excellence for MIPLYFFA and OLPRUVA while advancing its clinical pipeline.
Differentiator
Problem solved
Functional benefit
Brands
- MIPLYFFA: U.S. FDA-approved treatment (arimoclomol) for neurological manifestations of Niemann-Pick disease type C (NPC) in adult and pediatric patients 2 years of age and older, used in combination with miglustat.
- OLPRUVA
Products and services
- MIPLYFFA (arimoclomol) First U.S. FDA-approved treatment for Niemann-Pick disease type C (NPC); an orally-delivered, first-in-class therapy indicated for use in combination with miglustat for the treatment of neurological manifestations of NPC in adult and pediatric patients 2 years of age and older.
- OLPRUVA (sodium phenylbutyrate) U.S. FDA-approved dual-coated sodium phenylbutyrate oral suspension for the treatment of certain patients living with urea cycle disorders (UCDs) involving deficiencies of carbamylphosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS).
- Celiprolol Investigational selective adrenoceptor modulator being evaluated in the Phase 3 DiSCOVER trial for the treatment of COL3A1-positive Vascular Ehlers-Danlos Syndrome (VEDS), enrolling 150 patients with up to 400 mg/day dosing. Believed to decrease mechanical stress on the vascular wall of large arteries and hollow organs.
Quantifiable outcome
- FY2025 revenue growth of 350% year-over-year to $106.5 million
- +6 more outcomes
Companies that use Zevra Therapeutics
Customer profileSegments5 records
Ideal customer profiles1 record
Zevra Therapeutics technology and API
TechnologyTechnology focussed No
API detail
- Has API
- No
- API docs
- API detail
Core technology
AI maturity
App detail
Feature4 records
Zevra Therapeutics partnerships and signals
Strategic signalPartnerships
Six partnerships are on record, tiered flagship and major.
- GeneDx Holdings Corp.flagshipGeneDx and Zevra Therapeutics announced a partnership in March 2026 to launch a sponsored genetic testing program for Niemann-Pick disease type C (NPC) in the US. The program leverages GeneDx's ExomeDx test and rare-disease genomic dataset (GeneDx Infinity) to help diagnose NPC more quickly and integrate data into a large genomic dataset, with Zevra providing financial support.
- Commave TherapeuticsmajorIn March 2026, Zevra Therapeutics sold its entire SDX portfolio, including AZSTARYS and KP1077, to Commave Therapeutics for $50 million. The transaction also resolves pending litigation between the two companies stemming from a 2019 license agreement, with Commave having initiated legal action in Delaware Chancery Court in 2024. Zevra also repaid its $63 million term loan in full prior to the agreement, leaving the company debt-free.
- UnipharflagshipIn December 2025, Zevra Therapeutics signed an exclusive distribution agreement with Uniphar to expand access to MIPLYFFA (arimoclomol) for Niemann-Pick Disease Type C patients outside of Europe. The agreement supports Zevra's US and EU regulatory strategies and aims to improve patient access globally.
- OrsiniflagshipIn September 2024, Zevra Therapeutics partnered with Orsini to become the exclusive specialty pharmacy provider for MIPLYFFA (arimoclomol), the first FDA-approved treatment for Niemann-Pick disease type C. The partnership aims to expand patient access to the therapy through specialty pharmacy distribution and reimbursement support in the United States.
- Acer Therapeutics, Inc.flagshipIn November 2023, Zevra completed the acquisition of Acer Therapeutics, Inc., including the U.S. FDA-approved OLPRUVA® for the treatment of certain urea cycle disorders, and celiprolol, an investigational product for the treatment of vascular Ehlers-Danlos syndrome. Through Acer's existing infrastructure, Zevra efficiently emerged as a commercial-stage organization and quickly launched OLPRUVA in January 2024.
- Orphazyme, A/SflagshipIn May 2022, Zevra (then KemPharm) acquired substantially all of the assets of Orphazyme, A/S, a Denmark-based company focused on neurological rare diseases. The acquisition included arimoclomol, a product candidate intended for the treatment of Niemann-Pick disease type C (NPC), and welcomed many of Orphazyme's team members. Arimoclomol became the basis for MIPLYFFA, the first FDA-approved NPC treatment.
Scale indicators13 records
Recent moves11 records
Expansion highlights6 records
Zevra Therapeutics competitors and assessment
Company assessmentDirect peers
- Ultragenyx Pharmaceutical: Commercial-stage rare disease biopharma with multiple approved and late-stage therapies for ultra-rare genetic disorders, comparable in commercial focus on specialty distribution, payer coverage strategies, and orphan pricing model.
- Mirum Pharmaceuticals: Focused commercial rare disease company with FDA-approved therapies for cholestatic liver diseases, sharing the specialty pharmacy distribution model and orphan disease commercial playbook.
- Travere Therapeutics: Commercial-stage rare disease biopharma with approved therapies for ultra-rare conditions (previously Retrophin), where Neil McFarlane served as COO; comparable in therapeutic area focus and rare disease commercial approach.
- Amicus Therapeutics: Commercial rare disease biopharma with Galafold for Fabry disease and Pombiliti+Opfolda for Pompe, comparable in patient identification, genetic testing partnerships, and ultra-rare specialty distribution model.
- Ionis Pharmaceuticals: RNA-targeted rare disease biopharma with multiple approved and late-stage neurological assets, comparable in CNS-focused rare disease development and platform-based drug development approach.
- BridgeBio Pharma: Clinical and commercial-stage rare genetic disease company with approved Attruby for transthyretin amyloid cardiomyopathy and pipeline in Mendelian diseases; comparable in rare disease commercial model and genetic testing-driven patient identification.
Broad incumbents
- BioMarin Pharmaceutical: Larger established rare disease biopharma with broad portfolio across multiple rare genetic conditions including enzyme replacement therapies; relevant given BioMarin executive Josh Schafer's background and overlap in rare metabolic disease commercial model.
- Swedish Orphan Biovitrum (Sobi): Large established European rare disease biopharma with broad portfolio in hematology, immunology, and specialty care; comparable in European rare disease commercial strategy relevant to Zevra's EMA MAA expansion.
- Alexion (AstraZeneca Rare Disease): Global leader in rare disease commercial execution with Soliris/Ultomiris franchise and broader AstraZeneca rare disease portfolio, comparable as the gold standard for ultra-rare specialty pharmacy distribution and orphan pricing.
Others
- Mallinckrodt Pharmaceuticals: Specialty pharma with autoimmune and rare disease focus including autoimmune neurology franchise; comparable through executive overlap (Joshua Schafer's prior GM role) and specialty distribution model.
Market position
Strengths4 records
Weaknesses4 records
Competitive moat6 records
Key risks5 records
Key highlights7 records
Customer concentration
Zevra Therapeutics social profiles
Digital presenceZevra Therapeutics financial estimates
Financial estimateRevenue estimate
Valuation estimate
Zevra Therapeutics leadership team
Management profileNumber of profiles
Profiles10 records
Zevra Therapeutics subsidiaries and ownership
Company hierarchySubsidiaries1 record
Zevra Therapeutics funding detail
Funding detailFunding overview
Funding rounds9 records
Investors4 records
Funding detail is available on the Subscription and Enterprise plan.Contact sales →
Zevra Therapeutics M&A and investment
M&A and investmentM&A1 record
Investments
M&A and investment is available on the Subscription and Enterprise plan.Contact sales →
Frequently asked questions about Zevra Therapeutics
What does Zevra Therapeutics do?
Zevra Therapeutics is a commercial-stage rare disease biopharmaceutical company that develops and commercializes therapies for ultra-rare diseases. Its portfolio includes two FDA-approved products: MIPLYFFA (arimoclomol), the first FDA-approved treatment for Niemann-Pick disease type C (NPC), and OLPRUVA (sodium phenylbutyrate) for urea cycle disorders (UCDs). The company also has a late-stage pipeline asset, celiprolol, in a Phase 3 trial for Vascular Ehlers-Danlos Syndrome (VEDS). Distribution is conducted via a specialty pharmacy (Orsini) in the US and an international distribution partner (Uniphar) outside Europe.
Is Zevra Therapeutics a public or private company?
Zevra Therapeutics is a public company. It is classified as public and is currently operating.
When was Zevra Therapeutics founded?
Zevra Therapeutics was founded in 2022. It employs 51 to 100 people.
Where is Zevra Therapeutics based?
Zevra Therapeutics is headquartered in Orlando, United States, in the North America region.
How does Zevra Therapeutics make money?
Four revenue lines are on record. Commercial product sales are the primary driver. The others are expanded Access Program (EAP) reimbursements, asset divestiture (one-time) and royalty income (third-party).
Who are Zevra Therapeutics's main competitors?
Direct peers on record are Ultragenyx Pharmaceutical, Mirum Pharmaceuticals, Travere Therapeutics, Amicus Therapeutics, Ionis Pharmaceuticals and BridgeBio Pharma. Broad incumbents are BioMarin Pharmaceutical, Swedish Orphan Biovitrum (Sobi) and Alexion (AstraZeneca Rare Disease). Mallinckrodt Pharmaceuticals is listed as an others.
Does Zevra Therapeutics have an API?
No public API is recorded for Zevra Therapeutics.
What industry is Zevra Therapeutics in?
Zevra Therapeutics's product category is Rare Disease Therapeutics. Its primary akta.pro industry code is HLAIAIAD, Rare Neurology & Neurodegenerative Disorder Therapies, with a secondary code of HLAIAIAL, Rare Gastroenterology & Hepatology Disorder Therapies. Its NAICS code is 325414 and its SIC code is 2834.