Research & Development
Orphazyme's arimoclomol receives Fast Track Designation from US FDA for treatment of ALS
22 May 2020 -

Orphazyme A/S (CPH:ORPHA.CO), a biopharmaceutical company pioneering Heat-Shock Protein response for the treatment of neurodegenerative orphan diseases, announced on Friday the receipt of Fast Track Designation from the US Food and Drug Administration (FDA) for the development of arimoclomol for the treatment of Amyotrophic Lateral Sclerosis (ALS).

Reportedly, this is the third Fast Track Designation that arimoclomol has received from the FDA.

Arimoclomol has also received Fast Track status from the FDA for the treatment of Niemann-Pick disease Type C (NPC) and sporadic Inclusion Body Myositis (sIBM).

Fast Track is a designation by the FDA of an investigational drug for expedited review to facilitate development of drugs which treat a serious or life-threatening condition and fill an unmet medical need. Fast Track status entails eligibility for Accelerated Approval and Priority Review if certain criteria are met, as well as Rolling Review, which means that a company can submit completed sections of its Biologic License Application (BLA) or New Drug Application (NDA) for review by the FDA, rather than waiting until every section is completed before the entire application can be reviewed.

Orphazyme initiated a phase 3 trial in August 2018 with arimoclomol in ALS. Topline results from this trial are expected in the first half of 2021.