Sept. 24, 2024 – The FDA has approved the first treatment for a rare genetic disorder often diagnosed in childhood that can cause damage to the nerves and brain and lead to organ failure over time. People with the condition live for about 13 years, on average.
Estimated to affect less than 1,000 people in the U.S., Niemann-Pick disease type C progressively impacts the abilities to speak, swallow, and walk or move independently.
The disease, which also affects mental functions like learning and memory, is a lysosomal storage disorder – characterized by a buildup of fatty substances like cholesterol that damage the body and brain.
The generic name for the new drug is arimoclomol, and it will be marketed under the brand name Miplyffa. It is approved for people ages 2 and up through a combination of FDA pathways -- including its fast-track and breakthrough designations, as well as a special program that rewards companies for new pediatric treatments.
Niemann-Pick disease type C, or 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,” said Janet Maynard, MD, MHS, director of the FDA’s Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine, in a statement. “The first-ever approval of a safe and effective drug option for NPC will undoubtedly support the essential medical needs of those suffering.”
In its approval, the FDA cited a clinical trial of Miplyffa that included 50 people with the condition ranging in age from 2 to 19 years old. Many in the trial were also taking another drug, called miglustat, and the new FDA approval calls for the two medicines to be taken together. Both are taken orally.
People taking Miplyffa with miglustat for 12 months halted disease progression, as measured by a scale for symptom severity, according to a news release by drugmaker Zevra Therapeutics.
In its approval announcement, the FDA characterized the treatment as resulting in a “slower disease progression.” As part of its application, Zevra Therapeutics also submitted data from a 48-month study following the initial trial, the company said.
In 2021, the FDA declined to approve Miplyffa. But in August, an FDA advisory committee voted 11-5 in support of a statement that the drug had been shown effective.
Those voting in support noted that there was an unmet need for such a treatment, there were no apparent safety concerns, and the available data was consistent. Dissenters suggested that the data “did not meet evidentiary standards” and called for additional study, plus more testing on animals and in laboratories to better evaluate how the drug worked, according to a meeting summary.
In its approval, the FDA noted that common side effects of Miplyffa include upper respiratory tract infection, diarrhea, and decreased weight.
Zevra Therapeutics announced that the drug will be available within 2 to 3 months.