Zevra Therapeutics Expands Global Access to MIPLYFFA Through Distribution Partnership with Uniphar
核心洞察
Zevra Therapeutics has executed an exclusive expanded access distribution agreement with Ireland-based Uniphar (搜索) to broaden access to MIPLYFFA (arimoclomol) for Niemann-Pick Disease Type C (搜索) patients in select territories outside Europe.
MIPLYFFA, used in combination with miglustat, is the only treatment shown to halt disease progression in NPC (搜索), with improvement seen at week 12 and durable effects lasting more than five years.
The drug is currently approved by the FDA and commercially available in the U.S., while a Marketing Authorization Application is under review by the European Medicines Agency (搜索).
Zevra Therapeutics announced on December 29, 2025, that it has executed an exclusive expanded access distribution agreement with Uniphar (搜索), an Ireland-based pharmaceutical services provider, to expand global access to MIPLYFFA® (arimoclomol) for patients with Niemann-Pick Disease Type C (搜索) (NPC (搜索)). The agreement enables NPC patients to access the therapy for reimbursed named patient supply in select territories outside of Europe.
Addressing Critical Unmet Medical Need
NPC (搜索) is an ultra-rare, relentlessly progressive genetic disorder that leads to premature mortality. MIPLYFFA, used in conjunction with miglustat, represents the only treatment shown to halt disease progression by addressing the underlying pathology of NPC. Clinical data demonstrates improvement at the first evaluation at week 12, with durable effects maintained 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 (搜索)," said Neil F. McFarlane, Zevra's President and Chief Executive Officer. "By leveraging Uniphar (搜索)'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."
Clinical Evidence and Regulatory Status
MIPLYFFA received FDA approval on September 20, 2024, and is currently commercially available in the United States. The drug increases the activation of transcription factors EB (TFEB) and E3 (TFE3 (搜索)), resulting in the upregulation of coordinated lysosomal expression and regulation (CLEAR) genes. Additionally, MIPLYFFA has been shown to reduce unesterified cholesterol in the lysosomes of human NPC (搜索) fibroblasts, though the clinical significance of these findings is not fully understood.
In the pivotal Phase 3 trial, MIPLYFFA halted disease progression compared to placebo over the one-year duration when measured by the NPC (搜索) Clinical Severity Scale, the only validated disease progression measurement tool for this condition. The extensive clinical development program has generated data from more than 270 NPC patients worldwide through Phase 2/3 clinical trials, Open-Label Extension studies, Expanded Access Programs, and pediatric sub-studies, representing the most comprehensive clinical development program in NPC to date.
Safety Profile and Administration
MIPLYFFA is indicated for use in combination with miglustat for treating neurological manifestations of NPC (搜索) in adult and pediatric patients 2 years of age and older. The drug is available in oral capsule strengths of 47 mg, 62 mg, 93 mg, and 124 mg.
Clinical trials revealed specific safety considerations, including hypersensitivity reactions such as urticaria and angioedema in some patients. In Trial 1, two patients (6%) reported both urticaria and angioedema, while one patient (3%) experienced urticaria alone within the first two months of treatment. The most common adverse reactions (≥15%) in MIPLYFFA-treated patients who also received miglustat were upper respiratory tract infection, diarrhea, and decreased weight.
Mean increases in serum creatinine of 10% to 20% compared to baseline were reported across clinical trials, occurring mostly in the first month of treatment but not associated with changes in glomerular function. These increases reversed upon MIPLYFFA discontinuation.
Strategic Partnership Benefits
Brian O'Shaunnessy, Chief Commercial Officer at Uniphar (搜索), commented on the partnership: "Uniphar is proud to partner with Zevra to expand global access to MIPLYFFA. 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 (搜索)."
Uniphar (搜索) brings over 57 years of experience and partnerships with more than 200 multinational clients, with a team of 3,500+ professionals across 180 countries. The company's unified platform spans medical affairs, regulatory strategy, market access, patient engagement, commercial services, distribution and supply chain management.
Regulatory Outlook
While MIPLYFFA is approved and commercially available in the United States, Zevra has submitted a Marketing Authorization Application to the European Medicines Agency (搜索) for evaluation of arimoclomol for NPC (搜索) treatment. The drug has also received Orphan Medicinal Product designation by the EMA for NPC treatment, which could facilitate the regulatory review process in Europe.