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Zevra Therapeutics

Rare disease drugmaker · based in Celebration, United States · founded 2006 · ticker ZVRA · zevra.com

Zevra Therapeutics works on 4 rare diseases we track, including Niemann-Pick Disease Type C, Niemann-Pick Disease and 2 more. It has 2 active clinical trials, and all of them are looking for volunteers now. It also has 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.

Zevra Therapeutics pipeline

Each row lists the diseases Zevra Therapeutics is studying at that stage, from the ones closest to approval down to the earliest work.

Approved
Drugs the FDA has approved.

Apadaz (benzhydrocodone and acetaminophen) · Miplyffa (arimoclomol citrate)

Phase 3
Large studies, usually the last step before a company asks the FDA for approval.
Phase 2
Mid-size studies that check whether a drug works and at what dose.

Medium-chain Acyl-CoA Dehydrogenase Deficiency recruiting

Zevra Therapeutics clinical trials

These are Zevra Therapeutics trials for the rare diseases we track, from ClinicalTrials.gov. Statuses there can lag behind company news, so confirm with the study team before you plan around one.

2 trials looking for volunteers now

Looking for volunteersPhase 2Recently updatedNCT06773026

Study of Sodium Phenylbutyrate (ACER-001) for the Treatment of Pediatric and Adults Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)

Tests Sodium phenylbutyrate

For 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.

Ages 4 Years+1 location
View full study
Looking for volunteersPhase 3Hasn't posted an update in over a yearNCT05432466

Clinical Trial to Compare the Efficacy of Celiprolol to Placebo in Patients With Vascular Ehlers-Danlos Syndrome

Tests ACER-002 (celiprolol) 200 mg BID, Placebo BID

For Vascular Ehlers-Danlos Syndrome

This is a prospective, Phase 3, randomized, double-blind, placebo-controlled efficacy study to evaluate celiprolol in patients genetically confirmed as COL3A1-positive vEDS using a decentralized clinical trial design.

Ages 15 Years - 64 Years1 location
View full study
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FDA-approved drugs from Zevra Therapeutics

From the openFDA database. Tap a drug for side effects, dosing and help paying.

Apadaz benzhydrocodone and acetaminophen

Approved Feb 23, 2018. Taken by mouth.

Miplyffa arimoclomol citrate

For Niemann-Pick Disease Type C. Approved Sep 20, 2024. Taken by mouth.

More about Zevra Therapeutics

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.

Where Zevra Therapeutics trials take place

The trials above run at 2 study sites in 1 country. Tap a country to see its states, cities and hospitals.

United States
2▼

Rare diseases Zevra Therapeutics works on

Each disease page lists every open trial, not only Zevra Therapeutics's, plus approved treatments.

Niemann-Pick Disease Type C

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...

How common: Approximately 1 per 100,000 to 1 per 150,000 live births; higher in certain populations

Niemann-Pick Disease

Niemann-Pick disease is a rare lysosomal storage disorder where the body cannot properly break down cholesterol and other lipids within cells.

How common: Type A: 1 in 250,000; Type B: 1 in 100,000 to 150,000; Type C: 1 in 100,000 to 150,000

Urea Cycle Disorders

Urea Cycle Disorders (UCDs) are a group of genetic conditions affecting the enzymes that convert toxic ammonia to urea for excretion.

How common: About 1 in 35,000 births in the United States, based on newborn screening and registry data (Summar et al., 2013); Ornithine Transcarbamylase (OTC) deficiency is most common form

Vascular Ehlers-Danlos Syndrome

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.

How common: At least 1 in 150,000 people, and genetic studies suggest it may be as common as about 1 in 20,000 (GeneReviews)

Where to get support

Patient groups can explain a diagnosis, point you to specialists and tell you about trials before they open. Zevra Therapeutics's own site is zevra.com, and our help paying for treatment page lists drugmaker programs and charity funds.

Questions about Zevra Therapeutics