CELEBRATION, Fla. — December 29, 2025 — Leads & Copy — Zevra Therapeutics, Inc. (NasdaqGS: ZVRA), a commercial-stage company focused on providing therapies for people living with rare diseases, has entered into an exclusive expanded access distribution agreement with Uniphar, an Ireland-based pharmaceutical services provider.
The agreement enables Niemann-Pick Disease Type C (NPC) patients to access MIPLYFFA® (arimoclomol) for reimbursed named patient supply in select territories outside of Europe.
NPC is an ultra-rare, relentlessly progressive, genetic disorder that leads to premature mortality. MIPLYFFA, used with miglustat, is the only treatment shown to halt disease progression by addressing the underlying pathology of NPC, with improvement seen at the first evaluation at week 12, and durable effect for more than five years.
Zevra’s President and Chief Executive Officer, Neil F. McFarlane, stated that the distribution agreement enables them to further their mission by expanding access and supporting a greater number of patients and families living with NPC. He added that by leveraging Uniphar’s leadership and infrastructure, they have an opportunity to initially address a select patient population, while continuing to prioritize their U.S. commercial launch and prepare for a potential EU approval.
Uniphar’s Chief Commercial Officer, Brian O’Shaunnessy, said Uniphar is proud to partner with Zevra to expand global access to MIPLYFFA. He believes their 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.
MIPLYFFA is approved by the U.S. Food and Drug Administration and is commercially available in the U.S. A Marketing Authorisation Application for the evaluation of arimoclomol for the treatment of NPC has been validated and is under review by the European Medicines Agency (EMA).
MIPLYFFA (arimoclomol) is Zevra’s approved therapy for the treatment of Niemann-Pick disease type C (NPC). Approved by the U.S. Food and Drug Administration on Sep. 20, 2024, MIPLYFFA (arimoclomol) increases the activation of the transcription factors EB (TFEB) and E3 (TFE3) resulting in the upregulation of coordinated lysosomal expression and regulation (CLEAR) genes. MIPLYFFA has also been shown to reduce unesterified cholesterol in the lysosomes of human NPC fibroblasts. 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 of the trial when measured by the only validated disease progression measurement tool, the NPC Clinical Severity Scale. MIPLYFFA has also received Orphan Medicinal Product designation by the European Medicines Agency (EMA) for the treatment of NPC.
The data generated for MIPLYFFA has shown long-term, meaningful clinical outcomes with more than 5 years of patient experience across more than 270 NPC patients worldwide through a Phase 2/3 clinical trial, Open-Label Extension (OLE) study, Expanded Access Programs (EAP), and a pediatric sub-study, which is the most expansive clinical development program in NPC to date. Zevra has submitted a Marketing Authorization Application to the European Medicines Agency for the evaluation of arimoclomol for the treatment of Niemann-Pick disease type C.
MIPLYFFA 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.
Hypersensitivity reactions such as urticaria and angioedema have been reported in patients treated with MIPLYFFA during Trial 1: two patients reported both urticaria and angioedema (6%) and one patient (3%) experienced urticaria alone within the first two months of treatment. Discontinue MIPLYFFA in patients who develop severe hypersensitivity reactions. If a mild or moderate hypersensitivity reaction occurs, stop MIPLYFFA and treat promptly. Monitor the patient until signs and symptoms resolve.
MIPLYFFA may cause embryofetal harm when administered during pregnancy based on findings from animal reproduction studies. Advise pregnant females of the potential risk to the fetus and consider pregnancy planning and prevention for females of reproductive potential.
Across clinical trials of MIPLYFFA, mean increases in serum creatinine of 10% to 20% compared to baseline were reported. These increases occurred mostly in the first month of MIPLYFFA treatment and were not associated with changes in glomerular function.
During MIPLYFFA treatment, use alternative measures that are not based on creatinine to assess renal function. Increases in creatinine reversed upon MIPLYFFA discontinuation.
The most common adverse reactions in Trial 1 (≥15%) in MIPLYFFA-treated patients who also received miglustat were upper respiratory tract infection, diarrhea, and decreased weight.
Three (6%) of the MIPLYFFA-treated patients had the following adverse reactions that led to withdrawal from Trial 1: increased serum creatinine (one patient), and progressive urticaria and angioedema (two patients). Serious adverse reactions reported in MIPLYFFA-treated patients were hypersensitivity reactions including urticaria and angioedema.
Arimoclomol is an inhibitor of the organic cationic transporter 2 (OCT2) transporter and may increase the exposure of drugs that are OCT2 substrates. When MIPLYFFA is used concomitantly with OCT2 substrates, monitor for adverse reactions and reduce the dosage of the OCT2 substrate.
Based on animal findings, MIPLYFFA may impair fertility and may increase post-implantation loss and reduce maternal, placental, and fetal weights.
The recommended dosage of MIPLYFFA, in combination with miglustat, in patients with an eGFR ≥15 mL/minute to <50 mL/minute is lower than the recommended dosage (less frequent dosing) in patients with normal renal function.
MIPLYFFA capsules for oral use are available in the following strengths: 47 mg, 62 mg, 93 mg, and 124 mg.
To report SUSPECTED ADVERSE REACTIONS, contact Zevra Therapeutics, Inc. at toll-free phone 1-844-600-2237 or FDA at 1 800-FDA-1088 or www.fda.gov/medwatch.
Investor Contact:
Nichol Ochsner
+1 (732) 754-2545
nochsner@zevra.com
Media Contact:
Julie Downs
+1 (508) 246-3230
jdowns@zevra.com
Source: Zevra Therapeutics, Inc.
