Zevra Therapeutics

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.

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

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

• Rare disease therapeutics
• Specialty pharmaceuticals
• CNS drug development
• Prodrug technology platform
• Orphan disease treatments

Headquarters

HQ city

Orlando

HQ country

United States

HQ region

North America

Offices3 records

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Personnel, Technology or R&D, Supply Chain, Marketing or Sales, Operations

Revenue model

1. 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
2. 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
3. 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
4. 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

Go-to-market motion5 records

Distribution channels5 records

Marketing channels7 records

Core 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

Segments5 records

Ideal customer profiles1 record

Technology focussed No

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature4 records

Partnerships

Six partnerships are on record, tiered flagship and major.

• GeneDx Holdings Corp.flagshipStrategic or Co-development Partner · 20 March 2026GeneDx 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 TherapeuticsmajorStrategic or Co-development Partner · 16 March 2026In 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.
• UnipharflagshipChannel Partner/ Reseller/ Distributor · 29 December 2025In 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.
• OrsiniflagshipChannel Partner/ Reseller/ Distributor · 1 September 2024In 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.flagshipStrategic or Co-development Partner · 1 November 2023In 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/SflagshipStrategic or Co-development Partner · 1 May 2022In 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

Direct 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

• Xx.com/zevratx
• Facebookfacebook.com/zevratherapeutics
• LinkedInlinkedin.com/company/zevratherapeutics

Revenue estimate

Valuation estimate

Number of profiles

Profiles10 records

Subsidiaries1 record

Funding overview

Funding rounds9 records

Investors4 records

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A1 record

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about Zevra Therapeutics