Sept. 26, 2024 – Within days of approving the first treatment for a rare genetic disorder, the FDA has approved another treatment for the same condition.
Known as Niemann-Pick disease type C (NPC), the condition is estimated to affect about 1 per million people in the United States. Often diagnosed in childhood, it can lead to problems with walking, speaking, swallowing, hand movements, vision, and tremors. Neurological damage can be severe, and patients live for about 13 years, on average.
“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 Janet Maynard, MD, MHS, director of the FDA’s Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine, in a statement. “This approval again demonstrates the FDA’s commitment to work with the scientific community to overcome the unique challenges that may arise with rare disease drug development.”
The new treatment is designed to help improve neurological symptoms in both adults and children with the disease -- which happens because of changes in the NPC1 or NPC2 gene that cause fats and cholesterol to get stuck in cells, damaging the brain, liver, and other organs.
The generic name for the new drug is levacetylleucine, and it will be marketed under the brand name Aqneursa. Its approval follows a phase III clinical trial, published in January in the New England Journal of Medicine. The trial involved 60 patients with neurological symptoms, both children (ages 4 and up) and adults. Results showed that patients who took Aqneursa for 12 weeks had noticeable improvements in their ability to walk, sit, maintain balance, and speak compared to those who took a placebo.
Along with granting approval, the FDA gave Aqneursa priority review and orphan drug designations, meaning it’s considered an important treatment for a rare condition.
Mallory Factor, CEO of IntraBio Inc., the company that makes Aqneursa, celebrated the approval as a significant step forward in helping NPC patients. He said that the company would make the treatment available to all who need it.
Common side effects include stomachaches, trouble swallowing, airway infections, and vomiting. The FDA also warned that Aqneursa may harm unborn babies, so women should inform their doctor if they are pregnant or planning to become pregnant while using this drug.
The FDA late last week approved the first drug to treat Niemann-Pick disease type C. That drug, known under the brand name Miplyffa, was approved for people ages 2 and over and will be available in the coming months.