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ABOUT US
Who We Are
We are a commercial-stage company focused on addressing unmet needs for the treatment of rare diseases. We have a diverse portfolio of products and product candidates, which includes clinical and commercial stage assets. We collaborate with our community to involve key thought leaders, physicians, patients, care partners and advocacy groups in our 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 implementing data-driven development and commercialization strategies, we are overcoming complex drug development challenges to make new therapies available to the rare disease community.
We have therapies approved in the U.S. for the treatment of Niemann-Pick disease type C (NPC), urea cycle disorders (UCDs), and a partnered program for attention deficit hyperactivity disorder (ADHD).
Our Background
In January 2022, KemPharm, Inc., a company that developed unique prodrugs using its Ligand Activated Therapy® technology platform, refocused their efforts on rare disease drug development and began to seek product candidates to treat rare diseases.
In May 2022, the Company acquired substantially all of the assets of Orphazyme, A/S, a Denmark-based company focused on neurological rare diseases, which included arimoclomol, a product candidate intended for the treatment of Niemann-Pick disease type C, or NPC, and welcomed many of its team members.
Timed with Rare Disease Day in 2023, we changed our name from KemPharm to Zevra Therapeutics, reflecting the Company’s intensified dedication to developing life-changing therapies for people living with rare diseases, who have limited or no treatment options.
In November of the same year, Zevra completed the acquisition of Acer Therapeutics, Inc., including 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 of 2024.1
In January 2024, the FDA acknowledged receipt of the resubmission of the New Drug Application for arimoclomol as an orally-delivered, first-in-class treatment for NPC. In August 2024, Zevra participated in the inaugural meeting of the Genetic Metabolic Diseases Advisory Committee to review arimoclomol; and, on September 20th, MIPLYFFA® (arimoclomol) became the first U.S. FDA approved treatment for NPC.2 In July 2025, Zevra completed the submission of a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for the evaluation of arimoclomol for the treatment of NPC with the goal of expanding access to NPC patients across the globe. The MAA has been validated by the EMA and is under review.
Today, Zevra’s priorities are guided by the Company’s strategic plan categorized under four actionable pillars: commercial excellence, pipeline and innovation, talent and culture, and corporate foundation. The Company strives to deliver life-changing treatments to people living with rare diseases with immediate focus on executing the commercial launches of MIPLYFFA and OLPRUVA, prudently investing in celiprolol and existing pipeline assets, and building a strong corporate foundation while making disciplined growth investments in the future.
1OLPRUVA is indicated 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). For more information visit: olpruvahcp.com
2MIPLYFFA is indicated for use in combination with miglustat for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adult and pediatric patients 2 years of age and older. For more information visit: miplyffa-hcp.com
