Zevra Inks Global Deal to Expand Lifeline for Rare Disease Patients

CELEBRATION, Fla. – December 29, 2025 – Zevra Therapeutics has executed a pivotal distribution agreement with global pharmaceutical services provider Uniphar, creating a crucial pathway for its recently approved drug, MIPLYFFA® (arimoclomol), to reach patients suffering from the ultra-rare and fatal genetic disorder, Niemann-Pick Disease Type C (NPC).

The deal establishes an exclusive framework for Uniphar to manage a reimbursed named patient supply program in select territories outside of the United States and Europe. This move signals a significant step toward addressing the profound unmet medical need for a patient population that often exists outside the reach of major pharmaceutical markets, offering a beacon of hope where few options have existed.

A Bridge to Desperate Patients

Niemann-Pick Disease Type C is a relentlessly progressive lysosomal storage disorder with a global prevalence estimated to be as low as one in every 286,000 individuals. The disease, caused by genetic mutations that disrupt intracellular cholesterol transport, leads to the toxic accumulation of lipids in the brain, liver, and spleen. Its symptoms are devastating and varied, including severe neurological decline, cognitive impairment, and organ damage, ultimately leading to premature death.

For families affected by NPC, the journey is often marked by a lengthy “diagnostic odyssey” and a desperate search for effective treatments. Until recently, care has been largely symptomatic. This new agreement aims to bridge the access gap for MIPLYFFA, which was approved by the U.S. Food and Drug Administration (FDA) on September 20, 2024. The drug is the only treatment shown to halt the progression of NPC when used with an existing therapy, miglustat, with clinical data demonstrating a durable effect for more than five years.

“There remains a clear unmet need within the rare disease community and this distribution agreement enables us to further our mission by expanding access and supporting a greater number of patients and families living with NPC,” said Neil F. McFarlane, Zevra’s President and Chief Executive Officer, in a statement.

This initiative operates under an Expanded Access Program (EAP), also known as a named patient supply program. Such programs provide a regulatory and logistical framework to supply therapies that are approved in one country but not yet commercially available in another, directly to patients with life-threatening conditions. By establishing a reimbursed program, Zevra and Uniphar are working to create a sustainable model that moves beyond compassionate use and into a structured system of access for eligible patients.

A Strategic Global Play

While the humanitarian impact is clear, the agreement also represents a shrewd strategic move for Zevra Therapeutics. By establishing a global footprint for MIPLYFFA through a named patient program, the company can generate early revenue and build market presence while it pursues broader regulatory approvals.

Currently, Zevra is prioritizing the commercial launch of MIPLYFFA in the United States, where it has already seen strong uptake with 66% of covered lives having market access. Simultaneously, the company is navigating the rigorous review process in Europe. A Marketing Authorisation Application (MAA) for arimoclomol is under review by the European Medicines Agency (EMA). This is a critical step, particularly as a previous MAA submitted by the drug's original developer, Orphazyme A/S, was withdrawn in 2022. Zevra has expressed confidence in its new, more extensive data package, which includes experience from over 270 NPC patients.

The partnership with Uniphar allows Zevra to de-risk its global strategy. It creates a foothold in key international territories, gathering real-world data and establishing relationships with healthcare providers and reimbursement bodies before a full commercial launch is even possible in those regions. This model has proven effective, as seen with a French EAP for arimoclomol that has already contributed to Zevra's revenue.

With the global market for NPC therapies projected to grow exponentially—some analysts predict it could exceed $900 million by 2033—this early market penetration could prove invaluable. “By leveraging Uniphar’s leadership and infrastructure, we have an opportunity to initially address a select patient population, while continuing to prioritize our U.S. commercial launch and prepare for a potential EU approval,” McFarlane added.

The Logistical Backbone of Hope

Delivering a specialized therapy to individual patients scattered across the globe is a monumental logistical challenge. This is where Uniphar’s role becomes central. The Ireland-based firm has over 57 years of experience and a vast global network, including state-of-the-art GMP facilities in the U.S., Europe, and the Asia Pacific region equipped for everything from ambient to cryogenic storage.

Managing a named patient supply program requires more than just shipping boxes. It involves navigating a complex web of international customs, import regulations, and cold-chain requirements to ensure the therapy arrives safely and maintains its integrity. Uniphar's expertise in handling temperature- and time-sensitive pharmaceuticals, combined with its experience in managing EAPs, provides the robust operational backbone necessary for the program's success.

“Uniphar is proud to partner with Zevra to expand global access to MIPLYFFA,” commented Brian O’Shaunnessy, Chief Commercial Officer at Uniphar. “We believe our proven expertise in global distribution and supply chain management combined with Zevra's innovative approach to rare disease therapies will help make a life-changing difference to patients living with NPC.”

A Breakthrough in Disease Modification

MIPLYFFA's importance stems from its unique mechanism and impressive clinical results. The drug is an orally administered therapy that increases the activation of key transcription factors, which in turn boosts the cell's natural lysosomal clearance system. In the pivotal Phase 3 trial, it was shown to halt disease progression over a one-year period when measured by the validated NPC Clinical Severity Scale.

Indicated for use in combination with miglustat for adult and pediatric patients two years and older, MIPLYFFA represents a fundamental shift from purely symptomatic treatment to disease modification. While the therapy carries important safety considerations, including the potential for hypersensitivity reactions and embryofetal toxicity, its approval and expanded availability mark a turning point for the NPC community.

This distribution agreement is a core component of Zevra’s broader strategy, which is deeply rooted in serving the rare disease community. The company is also advancing celiprolol in a Phase 3 trial for Vascular Ehlers-Danlos Syndrome (vEDS), another rare condition with no approved treatments. For families navigating the relentless progression of Niemann-Pick Disease Type C, this logistical and strategic alliance represents a tangible expansion of hope, turning a once-distant therapy into a potential reality.