Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a commercial-stage company focused on providing therapies for people living with rare disease, announced that the Company has executed an exclusive expanded access distribution agreement with Uniphar, an Ireland-based pharmaceutical services provider with a proven record of success in global warehousing, distribution and supply chain management. 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 in conjunction 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.

“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. “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.”

Commenting on the agreement, Brian O’Shaunnessy, Chief Commercial Officer at Uniphar, said, “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.”

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).