The Food and Drug Administration (FDA) has granted Fast Track designation to arimoclomol (Orphazyme A/S) for the treatment of amyotrophic lateral sclerosis (ALS).

Arimoclomol is an investigational oral drug candidate that works by amplifying the production of heat-shock proteins (HSPs). The Company believes that HSPs may rescue defective misfolded proteins, clear protein aggregates and improve the function of lysosomes to treat ALS.

The Company is conducting an ongoing multicenter, placebo-controlled phase 3 trial of arimoclomol in 213 adult patients with ALS. Patients were randomized 2:1 to receive arimoclomol or placebo; the trial will include up to an additional 18 US patients on stable treatment with edaravone. The primary end point is the combined assessment of function and survival over 76 weeks. 

Results from the trial are expected in the first half of 2021. Additionally, patients who have completed the trial will be able to participate in an open-label extension trial.

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“This is the third Fast Track Designation that arimoclomol has received from the FDA, which further underlines the potential of our investigational drug, the seriousness, and high unmet medical need in the diseases that we are targeting,” said Kim Stratton, CEO, Orphazyme A/S.

The investigational drug is also being evaluated for the treatment of Niemann-Pick disease Type C (NPC), sporadic inclusion body myositis (sIBM), and Gaucher disease.

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