Zevra Inks Deal to Expand Global Access for Rare Disease Drug MIPLYFFA®

CELEBRATION, FL – December 29, 2025 – In a significant move to broaden the availability of a breakthrough therapy, Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) has executed an exclusive distribution agreement with the Ireland-based pharmaceutical services provider Uniphar. The partnership, announced today, is set to expand access to MIPLYFFA® (arimoclomol) for patients suffering from the ultra-rare and devastating Niemann-Pick Disease Type C (NPC) in select territories outside of the United States and Europe.

This strategic alliance leverages Uniphar’s extensive global logistics network to facilitate a reimbursed named patient supply program, offering a new lifeline to families affected by NPC in parts of the world where treatment options have been non-existent. The agreement underscores a critical step in addressing the profound unmet medical needs within the rare disease community.

The Fight Against a Relentless Disease

Niemann-Pick Disease Type C is an unforgiving, relentlessly progressive genetic disorder that disrupts the body's ability to transport cholesterol and other lipids within cells. This dysfunction leads to the accumulation of these substances in various tissues, including the brain, spleen, and liver, causing a cascade of severe neurological and systemic symptoms. The disease, which often appears in childhood, is invariably fatal, leading to premature mortality and immense suffering for patients and their families.

For years, the NPC community has grappled with a scarcity of effective treatments. The announcement highlights MIPLYFFA®, used in combination with miglustat, as a pivotal advancement. According to Zevra, it is the only treatment that has demonstrated an ability to halt the progression of the disease. Clinical data has shown measurable improvements as early as 12 weeks into treatment, with a durable effect lasting for more than five years, offering a degree of stability previously thought unattainable.

“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,” stated Neil F. McFarlane, President and Chief Executive Officer of Zevra. This sentiment captures the urgency and patient-centric focus driving the new partnership.

A Strategic Alliance to Bridge the Gap

The collaboration between Zevra and Uniphar is a calculated move designed for maximum impact with logistical efficiency. Zevra, a commercial-stage company, can maintain its strategic focus on its primary commercial launch in the United States and its preparations for a potential marketing approval in the European Union. Simultaneously, by partnering with Uniphar, it can address the immediate needs of patients in other regions without having to build a global distribution infrastructure from the ground up.

Uniphar brings to the table a proven track record in global warehousing, distribution, and supply chain management, making it an ideal partner for navigating the complexities of international pharmaceutical access. “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.

This sentiment was echoed by Uniphar’s Chief Commercial Officer, Brian O’Shaunnessy. “Uniphar is proud to partner with Zevra to expand global access to MIPLYFFA,” he commented. “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.”

Unpacking the Science of a Breakthrough Therapy

MIPLYFFA® received approval from the U.S. Food and Drug Administration (FDA) on September 20, 2024, and is already commercially available in the U.S. The therapy's mechanism of action is novel, working by increasing the activation of transcription factors TFEB and TFE3. This, in turn, upregulates a network of genes involved in coordinated lysosomal expression and regulation (CLEAR), effectively boosting the cell's natural 'cleanup' machinery to help clear the harmful accumulation of lipids characteristic of NPC.

The drug's approval was supported by a robust clinical development program, which Zevra describes as the most expansive in NPC to date. The pivotal Phase 3 trial demonstrated that MIPLYFFA halted disease progression compared to a placebo over a one-year period, as measured by the NPC Clinical Severity Scale—the only validated tool for measuring the disease's progression. The therapy's effectiveness and safety have been studied in over 270 NPC patients worldwide through clinical trials, open-label extension studies, and expanded access programs, gathering more than five years of patient experience.

While already approved in the U.S., a Marketing Authorisation Application for arimoclomol is currently under review by the European Medicines Agency (EMA), signaling a potential future expansion of its availability within Europe.

Understanding the Treatment's Safety Profile

As with any potent therapy, a comprehensive understanding of its safety profile is essential for patients and clinicians. The information provided by Zevra includes important safety considerations for MIPLYFFA. Hypersensitivity reactions, including urticaria (hives) and angioedema (swelling), have been reported. The company advises discontinuation of the drug in patients who experience severe reactions.

Based on findings from animal reproduction studies, the drug may cause embryofetal harm, and pregnancy planning and prevention are recommended for females of reproductive potential. Another notable finding from clinical trials was a mean increase in serum creatinine of 10% to 20%, which was not associated with changes in kidney function and reversed upon discontinuation of the treatment. Physicians are advised to use alternative measures to assess renal function during treatment.

The most common adverse reactions observed in the pivotal trial for patients taking MIPLYFFA in combination with miglustat were upper respiratory tract infection, diarrhea, and decreased weight. The partnership with Uniphar will not only distribute the therapy but also ensure that this vital safety and administration information is clearly communicated to treating physicians in new territories, ensuring patient well-being remains the highest priority as access expands.

This new chapter for MIPLYFFA® represents a convergence of scientific innovation and strategic logistics, aimed squarely at a patient population that has long awaited a meaningful therapeutic intervention. The agreement is a testament to a modern approach in the rare disease space, where collaboration is key to transforming clinical breakthroughs into tangible, accessible hope for patients around the world.