Zevra Therapeutics works on 4 rare diseases tracked on Trial Friend, including Niemann-Pick Disease Type C, Niemann-Pick Disease, Urea Cycle Disorders and 1 more, with 2 recruiting clinical trials and 2 FDA-approved rare disease drugs.
Zevra Therapeutics is a Florida-based rare disease company, renamed from KemPharm in February 2023 to mark its pivot to rare and ultra-rare conditions. Miplyffa (arimoclomol), approved in September 2024, was the first FDA-approved treatment for Niemann-Pick disease type C. Through its 2023 acquisition of Acer Therapeutics, Zevra also markets Olpruva (sodium phenylbutyrate) for certain urea cycle disorders and is running the Phase 3 DiSCOVER trial of celiprolol (Edsivo) for vascular Ehlers-Danlos syndrome under a Special Protocol Assessment with the FDA.
Zevra Therapeutics has 2 active clinical trials across 2 development stages, with 2 currently recruiting participants. Clinical trials advance through phases: Phase 1 tests safety in a small group, Phase 2 evaluates effectiveness and side effects, Phase 3 confirms benefit in a larger population, and Phase 4 monitors long-term safety after FDA approval.
Note: This pipeline counts the active and recruiting trials listed on this page, from ClinicalTrials.gov. For companies that work outside rare disease, we list only trials for rare diseases we track, so the company's full pipeline may be larger.
Active and recruiting clinical trials sponsored by Zevra Therapeutics, sourced live from ClinicalTrials.gov. Each trial card shows the study phase, current recruitment status, conditions under investigation, study locations, eligibility criteria, and a direct link to the full ClinicalTrials.gov record. You can also download a one-page PDF summary to share with your doctor.
Note: Recruitment statuses on ClinicalTrials.gov may not immediately reflect recent FDA decisions, sponsor announcements, or enrollment changes. Always confirm a trial's current status directly with the study coordinator.
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RECRUITINGPHASE2Updated a few months agoNCT06773026
Study of Sodium Phenylbutyrate (ACER-001) for the Treatment of Pediatric and Adults Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)
Intervention:Sodium phenylbutyrate
Medium-chain Acyl-CoA Dehydrogenase Deficiency
This is a medical research study to test a medication in patients 4 years of age and older with a disease called medium-chain acyl-CoA dehydrogenase deficiency (MCADD) caused by the common ACADM c.985 A\>G (K304E) mutation. The medication is sodium phenylbutyrate (ACER-001), which is currently FDA approved for the treatment of Urea Cyle Disorders. Previous research suggests that sodium phenylbutyrate may also be effective in the treatment MCADD. This study will investigate the safety and efficacy (how well it works) of sodium phenylbutyrate in patients with MCADD.
This is a prospective, Phase 3, randomized, double-blind, placebo-controlledefficacy study to evaluate celiprolol in patients genetically confirmed as COL3A1-positive vEDS using a decentralized clinical trial design.
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Zevra Therapeutics FDA-Approved Drugs (2)
Medications developed or marketed by Zevra Therapeutics that have received U.S. Food and Drug Administration approval. Drug data is sourced from the openFDA database and includes brand names, generic names, approval dates, and matched rare disease indications. Where a drug treats a condition covered by Trial Friend, the disease name links directly to that disease page.
Zevra Therapeutics clinical trials are running at 2 sites in 1 country. Click any country to drill down by state, city, and individual research facility. Proximity to a trial site is one of the most important factors in deciding whether to participate.
United States
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Rare Disease Focus Areas (4)
Diseases targeted by Zevra Therapeutics's clinical trial and drug development programs
Pharmaceutical and biotech companies working on similar rare diseases. Overlapping programs can mean more treatment options and competing trials for patients with these conditions.
Frequently Asked Questions About Zevra Therapeutics
Common questions about Zevra Therapeutics's rare disease programs, clinical trials, and treatments.
Zevra Therapeutics is a Florida-based rare disease company, renamed from KemPharm in February 2023 to mark its pivot to rare and ultra-rare conditions. Miplyffa (arimoclomol), approved in September 2024, was the first FDA-approved treatment for Niemann-Pick disease type C. Through its 2023 acquisition of Acer Therapeutics, Zevra also markets Olpruva (sodium phenylbutyrate) for certain urea cycle disorders and is running the Phase 3 DiSCOVER trial of celiprolol (Edsivo) for vascular Ehlers-Danlos syndrome under a Special Protocol Assessment with the FDA.
Zevra Therapeutics has active programs across 4 rare diseases: Niemann-Pick Disease Type C, Niemann-Pick Disease, Urea Cycle Disorders, Vascular Ehlers-Danlos Syndrome. Each disease page on Trial Friend shows the latest recruiting trials, FDA-approved treatments, and patient resources.
Yes. Zevra Therapeutics is a rare disease specialist headquartered in Celebration, United States, founded in 2006. The company focuses primarily on developing treatments for rare and orphan diseases, with programs targeting Niemann-Pick Disease Type C, Niemann-Pick Disease, Urea Cycle Disorders among others.
The clinical trials section on this page shows active and recruiting rare disease trials sponsored by Zevra Therapeutics, pulled live from ClinicalTrials.gov. Each trial card includes the study phase, recruitment status, conditions being studied, number of trial sites, and a direct link to the full study record. You can also explore individual disease pages to see trials from all sponsors for a specific condition.
To explore enrollment, find a recruiting trial in the clinical trials list on this page and click "View full study" to see the full ClinicalTrials.gov record, which includes eligibility criteria, study locations, and contact information for each trial site. Your doctor can also help determine if a specific trial is appropriate for your condition. Not all trials are open at every location, so check the site list for one near you.
Zevra Therapeutics has 1 FDA-approved drug with a rare disease use that we track, as of October 2026.
Source: the FDA's openFDA label database, matched to the rare diseases on Trial Friend; the list updates automatically and can include older products.
Zevra Therapeutics was founded in 2006 and is headquartered in Celebration, United States. The company has been active for 20 years. It trades publicly under the ticker symbol ZVRA. As a rare disease specialist, the company dedicates its resources to developing therapies for patients with rare and orphan conditions.