The U.S. Food and Drug Administration approved Miplyffa (arimoclomol), an oral medication for the treatment of Niemann-Pick disease, type C (NPC).
Miplyffa, in combination with the enzyme inhibitor miglustat, is approved to treat neurological symptoms associated with NPC in adults and children 2 years of age and older. Miplyffa is the first drug approved by the FDA to treat NPC.
The safety and effectiveness of Miplyffa were evaluated in a randomized, double-blind, placebo-controlled 12-month trial in patients two to 19 years of age who had a molecularly confirmed diagnosis of NPC. Fifty patients were randomized 2:1 to treatment with weight-adjusted Miplyffa (31 to 124 mg) or placebo orally three times per day. Among these 50 patients, 39 (78%) received miglustat as background treatment in the trial.
Miplyffa’s efficacy was demonstrated by the rescored 4-domain NPC Clinical Severity Scale (R4DNPCCSS) score in the patients who used miglustat as their background treatment. The R4DNPCCSS is a measure of NPC disease progression that looks at four items that patients with NPC, their caregivers and physicians have identified as most relevant including ambulation, speech, swallow and fine motor skills. Higher scores signify a greater severity of the disease. Compared to placebo, Miplyffa resulted in a slower disease progression as measured by the R4DNPCCSS score.
The prescribing information for Miplyffa contains a warning for hypersensitivity reactions including hives and angioedema (swelling under the skin). Individuals experiencing these adverse reactions should stop using the drug. Females who are pregnant or plan to become pregnant should not use Miplyffa.
The most common side effects of Miplyffa include upper respiratory tract infection, diarrhea and decreased weight.
Miplyffa, along with miglustat, should be taken orally with or without food according to the recommended dose for the patient’s body weight.
The FDA granted Miplyffa priority review, orphan drug, rare pediatric disease, fast track and breakthrough therapy designations for this application.
The FDA granted approval of Miplyffa to Zevra Therapeutics.
