FDA Approves Second Drug to Treat Rare Neurodegenerative Disease

NPC © syahrir - stock.adobe.com

The FDA approved IntraBio Inc.'s Aqneursa (levacetylleucine) yesterday for the treatment of ultra rare neurodegenerative disease Niemann-Pick Disease, Type C (NPC), according to a news release. This makes Aqneursa the second NPC drug approved within a week and the first standalone FDA-approved NPC treatment. Miplyffa gained approval last Friday.

NPC is a rare, progressive lysosomal storage disorder cause by mutations in the NPC1 and NPC2 genes. This leads to a buildup of lipids like cholesterol in cells, which leads to eventually fatal organ damage. As a result, NPC patients only live to about age 13. There are approximately 300 patients in the United States that have been diagnosed with NPC, but it’s estimated there are at least another 600 who remain undiagnosed.

Aqneursa is approved for patients who weigh at least 30 pounds. It comes as an oral suspension that can mixed into 40 mL of water, orange juice or almond milk if taken by mouth. If injected into a gastronomy tube, it must only be mixed with water. Aqneursa can be taken up to three times daily without or without food.

Safety and efficacy were determined during a IB1001-301 Phase 3 trial of 60 patients who were at least 4 years old with a confirmed NPC diagnosis and mild neurological symptoms such as double vision and ataxia. Patients were treated with Aqneursa for 12 weeks and placebo for 12 weeks. Order of treatments varied by patient. Full results were published in the the February 1, 2024 issue of the New England Journal of Medicine.

Efficacy was calculated using the Scale for the Assessment and Rating of Ataxia (SARA), that measured gait, sitting, stance and speech disturbance. On average, patients treated with Aqneursa had a better score than when they were treated with placebo and symptoms improvements were seen by week 12.

The most common adverse reactions were abdominal paid (2 patients), dysphagia (difficulty swallowing, 2 patients), upper respiratory tract infections (5 patients) and vomiting (2 patients).

Aqneursa may cause embryo-fetal harm if used during pregnancy. Patients who are pregnant or suspect they may be pregnant should tell their provider before starting Aqneursa.

Aqneursa is available now. Patients seeking support can sign up for Aqneursa Cares where they can find financial support, educational resources and access to a team of specialists.