Zevra Therapeutics
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 Drug Pipeline
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 includes all of Zevra Therapeutics's active interventional trials, not only those targeting rare diseases. We show the full pipeline because a company's broader research activity, therapeutic expertise, and development infrastructure directly shape its ability to advance rare disease programs. A strong overall pipeline often signals deeper clinical operations, faster enrollment capabilities, and greater commitment to bringing new treatments to patients.
Zevra Therapeutics Clinical Trials (2)
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.
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.
| Drug Name | Brand Name | Rare Diseases | Approval Date |
|---|---|---|---|
| BENZHYDROCODONE AND ACETAMINOPHEN | APADAZ oral | — | Feb 23, 2018 |
| ARIMOCLOMOL CITRATE | MIPLYFFA oral | Sep 20, 2024 |
Zevra Therapeutics Trial Locations
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.
Rare Disease Focus Areas (4)
Diseases targeted by Zevra Therapeutics's clinical trial and drug development programs
Niemann-Pick Disease Type C (NPC) is a rare, progressive lysosomal lipid storage disorder caused by mutations in the NPC1 or NPC2 genes, which encode proteins critical for cholesterol and lipid traffi...
Niemann-Pick disease is a rare lysosomal storage disorder where the body cannot properly break down cholesterol and other lipids within cells. These fatty substances accumulate in the liver, spleen, b...
Urea Cycle Disorders (UCDs) are a group of genetic conditions affecting the enzymes that convert toxic ammonia to urea for excretion. Deficiency of any of the eight enzymes in the urea cycle leads to ...
Vascular EDS is a rare genetic disorder affecting connective tissue that causes fragility in arteries and internal organs, making people vulnerable to life-threatening ruptures and bleeding. People wi...
Patient Resources
Organizations and resources related to Zevra Therapeutics's rare disease focus areas
Frequently Asked Questions About Zevra Therapeutics
Common questions about Zevra Therapeutics's rare disease programs, clinical trials, and treatments.