IntraBio Gets FDA Approval for Ultra-Rare Neurodegenerative Disease After Competitor

IntraBio’s Aqneursa has gained regulatory approval from the FDA for the treatment of Niemann-Pick disease type C (NPC), marking it as the second drug approved for this condition within a week, following closely behind Zevra Therapeutics’ Miplyffa. The FDA's clearance on Tuesday allows the use of Aqneursa, or levacetylleucine, to alleviate neurological symptoms in both adults and children who weigh at least 15 kg.

The approval of Aqneursa is based on data from a rigorous clinical trial that was randomized, double-blinded, and placebo-controlled. This crossover study demonstrated that patients treated with Aqneursa over a 12-week period exhibited improved functional performance, particularly in areas such as gait, sitting, stance, and speech. Additionally, the medication was found to be generally well-tolerated by patients. Common side effects noted included vomiting, abdominal pain, difficulty swallowing, and upper respiratory tract infections. The drug’s label also carries a warning regarding potential embryo-fetal harm if used during pregnancy.

Details regarding the cost and launch date of Aqneursa remain undisclosed.

Niemann-Pick disease type C is an ultra-rare neurodegenerative lysosomal storage disorder, affecting approximately one in every 150,000 individuals. It stems from mutations in the NPC1 and NPC2 genes, which disrupt the normal process of clearing cholesterol and other lipids from cells, resulting in their harmful accumulation in the brain and other vital organs. The condition significantly impairs speech, motor skills, and swallowing functions, with patients typically living only up to around 13 years of age.

The prompt approval of Aqneursa underscores the FDA's dedication to advancing the development of innovative treatments for rare diseases. Janet Maynard, director of the FDA’s Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine, emphasized the regulator’s commitment to supporting such medical advancements in a statement.

Just days before Aqneursa’s approval, Zevra Therapeutics received the FDA’s nod for its oral drug Miplyffa (arimoclomol), also targeting the neurological symptoms of NPC. Miplyffa is to be taken in conjunction with miglustat and is suitable for adults and children aged two years and older. Similar to Aqneursa, Miplyffa can be administered up to three times daily, with dosing schedules tailored to the patient’s body weight.

Zevra has disclosed plans to price Miplyffa between $40,000 and $106,000 per month, depending on the dosage, with an expected market launch in eight to 12 weeks.