Posted by Michael Wonder on 20 Jul 2020
Orphazyme completes rolling submission of new drug application to U.S. FDA for arimoclomol for Niemann-Pick disease type C
20 July 2020 - Company expects to submit marketing authorisation application for arimoclomol for Niemann-Pick disease type C to European Medicines Agency in the second half of 2020.
Orphazyme today announced that it has completed the rolling submission of its new drug application to the U.S. FDA for its investigational therapy arimoclomol for the treatment of Niemann-Pick disease type C.
Arimoclomol has received FDA fast track and breakthrough therapy designations for Niemann-Pick disease type C, as well as orphan drug and rare paediatric disease designations.
