Arimoclomol Prospective Double-blind, Randomised, Placebo-controlled Study in Patients Diagnosed With Niemann-Pick Disease Type C
Latest Information Update: 25 Apr 2025
At a glance
- Drugs Arimoclomol (Primary)
- Indications Niemann-Pick disease type C
- Focus Registrational; Therapeutic Use
- Sponsors Orphazyme; Zevra Therapeutics
- 21 Apr 2025 According to a Zevra Therapeutics Media release, the company has recently sold the Rare Pediatric Disease Priority Review Voucher (PRV) for $150 million to further enhance financial flexibility.
- 30 Jan 2025 According to a Zevra Therapeutics Media release, data from the study has been accepted for the presentation at the 21st Annual WORLDSymposium™.
- 28 Nov 2024 Status changed from active, no longer recruiting to completed.