IntraBio’s Aqneursa is the second drug within a week approved by the regulator for treating Niemann-Pick disease type C, just days behind Zevra Therapeutics’ Miplyffa.
The FDA on Tuesday signed off on IntraBio’s levacetylleucine for the treatment of Niemann-Pick disease type C. The drug, which will now be marketed under the brand name Aqneursa, is indicated to ease neurological symptoms in both adults and children weighing at least 15 kg.
Aqneursa was backed by data from a randomized, double-blinded and placebo-controlled crossover study. During the 12-week period that patients were treated with Aqneursa, they showed better functional performance as measured by a score that assesses their gait, sitting, stance and speech.
The study also found Aqneursa to be well-tolerated, with common side effects being vomiting, abdominal pain, difficulty swallowing and upper respiratory tract infections. The drug’s label includes a warning for embryo-fetal harm if used during pregnancy, according to the FDA’s announcement.
It remains unclear how much Aqneursa will cost or when it will launch.
Afflicting approximately one in every 150,000 people, Niemann-Pick disease type C (NPC) is an ultra-rare and neurodegenerative lysosomal storage disease that involves the progressive impairment of patients’ physical and cognitive function. Mutations in the NPC1 and NPC2 genes compromise the body’s otherwise healthy mechanism of clearing cholesterol and other lipids from inside cells, leading to their pathologic build-up in the brain and in other crucial organs.
NPC affects patients’ speech, motor and swallowing functions. According to the FDA, patients with NPC typically live only up to around 13 years.
Aqneursa’s approval on Tuesday marks the second NPC approval in a week, highlighting the regulator’s “commitment” to supporting the development of innovative therapies for rare diseases, Janet Maynard, director of the FDA’s Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine, said in a statement.
OnFriday, the regulator greenlit Zevra Therapeutics’ oral drug Miplyffa (arimoclomol), also for the treatment of the neurological manifestations of NPC. Miplyffa is meant to be taken with miglustat and can be administered to adults and children aged two years and older. Like Aqneursa, Miplyffa can be given up to three times per day, with or without food, with the exact dosing schedule depending on the patients’ body weight.
Zevra in a conference call on Monday announced that it would price Miplyffa between $40,000 to $106,000 per month, depending on dosage, according to Reuters. The company expects to launch Miplyffa in eight to 12 weeks.
