FDA approves Miplyffa for Niemann-Pick disease type C

The FDA announced in a press release that it has approved Miplyffa to treat neurological symptoms associated with Niemann-Pick disease type C in patients aged 2 years and older.

Miplyffa (arimoclomol; Zevra Therapeutics), the first drug approved to treat the condition, is an oral therapy that can be administered three times a day with or without food, by either the patient or caregiver, according to a press release from the manufacturer. Precise dosing, ranging from 47 mg to 124 mg, will be dependent upon the patient’s body weight, with follow up by a health care provider, the company said.

“The approval of Miplyffa is a monumental milestone for patients and their family members,” Neil F. McFarlane, president and CEO of Zevra Therapeutics, said in the release.

“We are immensely grateful for the unwavering support we have received over the years from the families and individuals impacted by [Niemann-Pick disease type C] as well as the collaborative efforts of advocacy groups, researchers and clinicians.”The FDA’s approval was based on safety and efficacy data from a 12-month multicenter, randomized, double-blind, placebo-controlled trial in patients aged 2 to 19 years with the rare and fatal neurodegenerative condition, which affects hundreds of individuals in the United States, according to the release from Zevra.

In the study, 76% of patients in the treatment group and 81% of those in the placebo group received miglustat, with efficacy measured by the 4-domain NPC Clinical Severity Scale (R4DNPCCSS) showing that Miplyffa with miglustat arrested disease progression by 12 months through a decrease of 0.2 points from baseline on the R4DNPCCSS compared with 1.9 points for those given miglustat alone. The FDA had previously granted the novel therapy priority review, orphan drug, rare pediatric disease, fast track and breakthrough therapy designations. Zevra further revealed it has received a rare pediatric disease priority review voucher in conjunction with the approval.

Zevra said it plans for Miplyffa to be commercially available in the U.S. in the next 8 to 12 weeks.

“[Niemann-Pick disease type C] 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,” Janet Maynard, MD, MHS, director of the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine in the FDA’s Center for Drug Evaluation and Research, said in the FDA release.

Reference:

FDA approves first treatment for Niemann-Pick disease, type C. https://www.fda.gov/news-events/press-announcements/fda-approves-first-treatment-niemann-pick-disease-type-c. Published Sept. 20, 2024. Accessed Sept. 20, 2024.