Within the span of a week, the FDA gave the green light to another drug for the rare condition Niemann-Pick disease type C in the form of IntraBio’s levacetylleucine.
On Friday, the FDA approved Zevra Therapeutics’ Miplyffa for NPC when added to Johnson & Johnson’s enzyme inhibitor Zavesca. Zevra had resubmitted the thrice-daily pill with a reanalysis of its registrational trial after its initial attempt at approval was rejected in 2021.
The approval of IntraBio’s levacetylleucine, which will be marketed as Aqneursa, was based on data from a 60-participant Phase 3 trial that showed the treatment achieved a “clinically meaningful” 1.37-point reduction on the Scale for the Assessment and Rating of Ataxia (SARA) score at 12 weeks versus placebo (p<0.001). SARA measures a patient’s level of muscle control when doing activities like walking, sitting and standing in place.
Aqneursa, which is a modified amino acid drug, is indicated for both adults and children weighing at least 15 kg. The oral treatment can be taken up to three times per day with dosing dependent on patient weight.
The back-to-back authorizations reflect the FDA’s “commitment to support development of new treatments for rare diseases,” Janet Maynard, director of CDER’s Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine, said in a Tuesday release.
Prior to these approvals, the only options to manage NPC were pain medications, physical therapy and speech therapy, among other strategies. Zavesca, which is approved for certain patients with Gaucher disease in the US, is also an off-label option.
Driven by changes in the NPC1 or NPC2 genes, NPC causes poor muscle tone, difficulty coordinating movements and progressive organ damage. There are around 300 to 350 people with the condition in the US, according to William Blair analysts.