Zevra Therapeutics has received the US Food and Drug Administration (FDA) approval for Miplyffa (arimoclomol) to treat Niemann-Pick disease type C (NPC).
Miplyffa is intended for use as an orally delivered treatment with miglustat to treat neurological symptoms in patients aged two and older.
The latest clearance makes Miplyffa the first approved drug for NPC in the US.
Additionally, Zevra Therapeutics has been awarded a rare paediatric disease priority review voucher (PRV) with this approval.
Previously, the drug received priority review, orphan drug, fast track, and breakthrough therapy designations.
FDA Center for Drug Evaluation and Research’s Office of Rare Diseases, Paediatrics, Urologic and Reproductive Medicine (ORPURM) director Janet Maynard said: “NPC is a serious disease that leads to enormous adverse impacts on patients and families. Despite extensive research efforts, there have not been approved treatments to meet the significant needs of patients.
“The first-ever approval of a safe and effective drug option for NPC will undoubtedly support the essential medical needs of those suffering.”
The FDA approval is based on comprehensive data in the new drug application (NDA). This included supporting evidence for trial endpoints and preferred FDA analyses.
Miplyffa’s safety and effectiveness were evaluated in a 12-month multicentre, randomised, double-blind, placebo-controlled trial involving patients aged two to 19.
In this study of 50 patients, 76% of arimoclomol patients, and 81% of the placebo group received miglustat as part of their care.
The effectiveness was measured using the rescored 4-domain NPC Clinical Severity Scale (R4DNPCCSS).
Results showed that Miplyffa, combined with miglustat, halted disease progression.
Patients on arimoclomol experienced a decrease of 0.2 points on the R4DNPCCSS, while those on miglustat alone had a progression of 1.9 points.
Additional evidence from a 48-month open-label extension study indicated improved outcomes compared to a matched NPC natural history cohort from the National Institutes of Health.
Zevra Therapeutics president and CEO Neil McFarlane said: “NPC is an ultra-rare, relentlessly progressive, degenerative, and fatal disease for which there were no FDA-approved treatment options until today.
“The approval of MIPLYFFA is a monumental milestone for NPC patients and their family members in the US.”
US-based Zevra Therapeutics plans to begin launch activities for Miplyffa immediately. The drug is expected to be commercially available in the US within eight to 12 weeks.
Concurrently, the company launched AmplifyAssist, a patient support programme.
