The FDA has granted approval for the first-ever treatment targeting Niemann-Pick disease type C (NPC), a rare genetic disorder that typically manifests in childhood and can lead to severe neurological damage and organ failure. Patients with NPC generally have a life expectancy of about 13 years.
Affecting an estimated fewer than 1,000 individuals in the U.S., Niemann-Pick disease type C disrupts the ability to speak, swallow, and move independently, while also impairing mental functions such as learning and memory. This lysosomal storage disorder is characterized by the accumulation of fatty substances, including cholesterol, which harm the body and brain.
The newly approved medication, arimoclomol, will be marketed under the brand name Miplyffa. It has received approval for individuals aged 2 and older through a combination of FDA designations, including fast-track and breakthrough statuses, as well as a special program aimed at encouraging new pediatric treatments.
Dr. Janet Maynard, director of the FDA’s Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine, stated, “NPC is a serious disease that leads to significant adverse effects on patients and their families. 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’s decision was informed by a clinical trial involving 50 participants aged 2 to 19, many of whom were also taking another medication called miglustat. The FDA has recommended that Miplyffa be used in conjunction with miglustat. Patients taking both medications for 12 months showed halted disease progression, as measured by symptom severity scales.
In its announcement, the FDA noted that the treatment results in “slower disease progression.” Zevra Therapeutics, the drug’s manufacturer, also submitted data from a 48-month follow-up study to support the application.
Initially, the FDA declined to approve Miplyffa in 2021. However, an advisory committee voted 11-5 in August in favor of the drug, citing the urgent unmet need for such a treatment and the absence of significant safety concerns.
Common side effects of Miplyffa include upper respiratory tract infections, diarrhea, and weight loss. Zevra Therapeutics has announced that the drug will be available within the next 2 to 3 months.
