Posted by Michael Wonder on 28 Dec 2023
Resubmission of arimoclomol new drug application to the US FDA
27 December 2023 - Zevra Therapeutics today announced it resubmitted its new drug application for arimoclomol, an investigational therapeutic candidate for the treatment of Niemann-Pick disease type C to the US FDA on 22 December 2023.
Zevra believes it has addressed the issues previously raised by the FDA in the complete response letter by providing additional evidence to support the use of the Niemann-Pick type C Clinical Severity Scale and, conducting additional studies used to support the potential mechanism of action.
