FDA Approves Treatments for Niemann-Pick Disease, Type C

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In late September 2024, FDA approved two drugs for the treatment of neurological symptoms associated with Niemann-Pick disease, type C (NPC) in both adults and children. According to FDA, patients with NPC experience progressive neurological symptoms and organ dysfunction caused by changes in the NPC1 or NPC2 gene that impact the transport of cholesterol and other lipids in a cell. This is a rare genetic condition, and patients with NPC may live only approximately 13 years (1).

Miplyffa (arimoclomol), approved on September 20, is the first treatment approved by FDA for NPC and is an oral medication used in combination with the enzyme inhibitor miglustat to treat NPC symptoms in adults and children two years of age and older. FDA’s Genetic Metabolic Diseases Advisory Committee discussed the application for Miplyffa at its first meeting in August 2024 (2, 3). The approval was based on results from a randomized double-blind, placebo-controlled 12-month trial that included patients aged two to 19 years of age. Of those patients, 50 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.” These studies showed that Miplyffa resulted in slower disease progression compared to placebo.

“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, director of the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine in the FDA’s Center for Drug Evaluation and Research, in a press release (1). “The first-ever approval of a safe and effective drug option for NPC will undoubtedly support the essential medical needs of those suffering.”

The second drug, Aqneursa (levacetylleucine), was approved on September 24 to treat NPC in adults and children who weight at least 15 kilograms. A randomized, double-blind, placebo-controlled, two-period, 24-week crossover study found that patients treated with Aqneursa for 12 weeks had better outcomes in the functional Scale for the Assessment and Rating of Ataxia (fSARA) compared with those that received a placebo. The fSARA evaluates “gait, sitting, stance, and speech disturbance domains of the original SARA with modifications to the scoring responses” (4).

“This is the second treatment the FDA has approved for NPC within the span of a week. Today’s action further underscores the agency’s commitment to support development of new treatments for rare diseases,” said Maynard, in a press release (4). “This approval again demonstrates the FDA’s commitment to work with the scientific community toovercome the unique challenges that may arise with rare disease drug development.”

References

1. FDA. FDA Approves First Treatment for Niemann-Pick Disease, Type C. Press Release. Sept. 20, 2024.
2. FDA. Updated Public Participation Information: August 2, 2024: Meeting of the Genetic Metabolic Diseases Advisory Committee Meeting Announcement. Aug. 2, 2024.
3. FDA. Genetic Metabolic Diseases Advisory Committee. FDA.gov (accessed Sept. 25, 2024).
4. FDA. FDA Approves New Drug to Treat Niemann-Pick Disease, Type C. Press Release. Sept. 24, 2024.