A US Food and Drug Administration (FDA) panel of experts has recommended approval of Zevra Therapeutics’ arimoclomol for Niemann-Pick disease type C (NPC), an ultra-rare neurodegenerative lysosomal storage disorder that affects one in every 100,000 to 120,000 births.
The Genetic Metabolic Diseases Advisory Committee voted 11 to five that the data submitted by Zevra supported the drug as an effective treatment option for the disease, which is caused by mutations in the NPC1 or NPC2 genes and characterised by the inability to properly metabolise lipids.
Patients progressively lose independence due to physical and cognitive limitations, with key neurological impairments presenting in speech, cognition, swallowing, ambulation and fine motor skills.
Administered orally, arimoclomol is designed to increase the production and activation of molecular chaperone proteins, which could increase the breakdown of accumulating lipids.
Among the data supporting the committee’s decision were long-term results from the arimoclomol expanded access programme, which were presented at this year’s Inherited Metabolic Disorders Annual Meeting in April.
Results showed that adults treated with arimoclomol, including those receiving and not receiving the substrate reduction therapy miglustat, generally had a stable disease course over two years of treatment and follow-up. The safety profile of the drug was also shown to be consistent with that observed in a phase 2/3 study.
Zevra’s president and chief executive officer, Neil McFarlane, said: “We are extremely pleased with the committee’s recognition of the benefits of arimoclomol for people living with NPC.
“Based on the totality of the clinical data, including data from the pivotal trial, the long-term data from the arimoclomol open-label extension study, and data from our expanded access programmes, we remain confident in the clinical benefit offered by arimoclomol as a treatment for NPC and are optimistic about its continued path to approval.”
The recommendation will now be considered by the FDA as it completes its independent review of arimoclomol, with a final decision expected by 21 September.
The drug has already been granted orphan drug, fast track, breakthrough therapy and rare paediatric disease designations by the FDA, as well as orphan medicinal product designation by the European Medicines Agency.