Zevra Therapeutics is focused on developing innovative therapies for rare diseases, leveraging a unique approach to drug development that prioritizes patient needs and regulatory narratives. The company has established a late-stage clinical pipeline that includes two partnered commercial products. One of their key programs is the development of Celiprolol, a new chemical entity aimed at treating Vascular Ehlers-Danlos Syndrome (VEDS), specifically for patients with COL3A1-positive VEDS. This program is currently in Phase 3 trials, having received both orphan drug and breakthrough therapy designations from the U.S. FDA, which underscores its potential significance in addressing unmet medical needs.

Another prominent candidate in Zevra's pipeline is KP1077, a prodrug of d-methylphenidate, which is being investigated for the treatment of idiopathic hypersomnia and narcolepsy. KP1077 has been granted orphan drug designation for idiopathic hypersomnia and is undergoing clinical trials to evaluate its safety and efficacy. The company has completed Phase 2 trials for idiopathic hypersomnia and Phase 1 trials for narcolepsy, with plans to analyze data from these studies to support future New Drug Applications. Zevra's commitment to addressing rare diseases is evident in their strategic focus on developing therapies that can significantly improve patient outcomes.

United States

We are a rare disease therapeutics company leading with science to make life-changing therapeutics available to patients with significant unmet needs. We involve key thought leaders, physicians, patients, care partners, and advocacy groups in all of our clinical and regulatory development strategies.

With a keen understanding that drug development often requires creative solutions, we have the insight and expertise to forge new pathways to success that others have missed. By following the data without bias, our transparent narratives and common-sense perspective have successfully overcome complex development challenges to make much-needed therapies available to patients.

Nimble and dauntless, we push boundaries beyond what is thought to be possible and advance new therapies that have the potential to bring meaningful improvement to patients’ lives.

Central Nervous System (CNS)

Genetic Diseases

Metabolic Diseases

Nervous System Diseases

Psychological Diseases

Zevra Therapeutics

Attention deficit hyperactivity disorder

Serdexmethylphenidate is a prodrug of dexmethylphenidate. Dexmethylphenidate HCl is a central nervous system (CNS) stimulant. The mode of therapeutic action in ADHD is not known.
<a href="https://kempharm.com/wp-content/uploads/2021/03/AZSTARYS-Master-Label-Final_20210302.pdf">Source</a>

AZSTARYS (KP415) (serdexmethylphenidate and dexmethylphenidate)

Acute pain

APADAZ (benzhydrocodone and acetaminophen)

Niemann Pick Disease Type C

Rare diseases

Rare genetic disease

A small molecule manufactured in a capsule formulation; designed to be swallowed whole, opened to allow contents to be mixed with soft foods/liquids or delivered through a nasal gastric tube or PEG Designed to cross the blood brain barrier to address neurodegenerative manifestations of disease
 
Arimoclomol is an investigational drug candidate that amplifies the production of Heat-Shock Proteins (HSPs). HSPs can rescue defective misfolded proteins, clear protein aggregates, and improve the function of lysosomes. Arimoclomol is administered orally, crosses the blood-brain barrier, and has now been studied in seven phase 1, four phase 2 and one pivotal phase 2/3 trial.0

MIPLYFFA™ (arimoclomol)

Vascular Ehlers-Danlos Syndrome

Celiprolol

EDS in Idiopathic Hypersomnia

Serdexmethylphenidate (SDX) is KemPharm’s proprietary prodrug of d-methylphenidate (d-MPH) and the sole active pharmaceutical ingredient (API) in KP1077, KemPharm’s lead clinical candidate being developed as a treatment for idiopathic hypersomnia (IH) and narcolepsy.

KP1077 (serdexmethylphenidate, SDX)

Stimulant Use Disorder

KP879 utilizes serdexmethylphenidate (SDX), KemPharm’s prodrug of d-MPH &nbsp;which is also the primary active pharmaceutical ingredient of KP415 and &nbsp;KP484, the company’s co-lead clinical development product candidates &nbsp;which are intended for the treatment of ADHD.
Based on information gathered during the KP415 and KP484 development &nbsp;programs, KemPharm believes that SDX has pharmacokinetic properties that &nbsp;are desirable as an agonist replacement therapy for the treatment of &nbsp;SUD. Specifically, KemPharm observed that SDX provided a gradual onset &nbsp;followed by long duration of d-MPH exposure, while also exhibiting the &nbsp;potential for a reduction in abuse-related effects.&nbsp;Given these &nbsp;properties, KemPharm is advancing KP879 as a potential first-ever &nbsp;therapeutic treatment for SUD.
<a href="https://kempharm.com/pipeline-products/#kp879">Source</a>

Zevra Therapeutics

Company Details