In a significant breakthrough for patients with Neimann-Pick disease type C (NPC), the FDA has approved Zevra Therapeutics, Inc.’s ZVRA Miplyffa (arimoclomol), an oral medication for this rare genetic disorder. Miplyffa, used in combination with Johnson & Johnson’s JNJ enzyme inhibitor Zavesca (miglustat), is approved to treat neurological symptoms associated with NPC in adults and children aged two years and older.
This marks the first FDA-approved drug specifically for treating NPC, offering a glimmer of hope for individuals battling this debilitating disease. NPC is characterized by progressive neurological symptoms and organ dysfunction, stemming from genetic mutations in either the NPC1 or NPC2 gene. These mutations disrupt the crucial transport of cholesterol and other lipids within cells, leading to cellular dysfunction and ultimately organ damage.
The safety and effectiveness of Miplyffa were rigorously evaluated in a 12-month randomized, double-blind, placebo-controlled trial involving patients with NPC. The trial demonstrated the efficacy of Miplyffa in slowing disease progression, as measured by the rescored 4-domain NPC Clinical Severity Scale (R4DNPCCSS). The R4DNPCCSS score is a reliable indicator of NPC disease progression, with higher scores reflecting greater severity. Patients receiving Miplyffa, in conjunction with their existing miglustat treatment, showed a slower disease progression compared to those receiving a placebo.
The path to Miplyffa’s approval involved a series of critical milestones. In August 2023, the inaugural meeting of the Genetic Metabolic Diseases Advisory Committee (GeMDAC) saw the first discussion of Miplyffa’s application. The committee, established in December 2023 to advise the FDA on products related to genetic metabolic diseases, voted favorably (11 yes, 5 no) that the data supported the effectiveness of arimoclomol in treating NPC patients.
Following a complete response letter from the FDA in 2021 requesting additional evidence supporting the validity and interpretation of the 5-domain NPC Clinical Severity Scale, particularly the swallow domain, Zevra Therapeutics resubmitted its New Drug Application (NDA) for arimoclomol in December 2023. This resubmission culminated in the FDA’s recent approval, signifying a major victory for Zevra Therapeutics and, most importantly, for the NPC community.
Following the approval announcement, ZVRA stock experienced a 1.01% increase, closing at $8.01 on Friday. This positive market response underscores the significance of this milestone for Zevra Therapeutics and the potential impact of Miplyffa on the lives of NPC patients.
