Arimoclomol Granted Fast Track Designation for Amyotrophic Lateral Sclerosis


Topline results from the phase 3 trial of arimoclomol are expected to be announced in the first half of 2021.

Kim Stratton

Kim Stratton

Orphazyme has announced that arimoclomol, its investigational drug candidate that amplifies the production of heat-shock proteins (HSPs), has been granted fast track designation by the FDA for the treatment of amyotrophic lateral sclerosis (ALS).1

“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. We are continuing to evaluate arimoclomol in a phase 3 clinical trial in ALS and look forward to topline results from the trial in [the first half of] 2021,” Kim Stratton, chief executive officer, Orphazyme, said in a statement.

A phase 3 trial (NCT03491462) for arimoclomol in patients with ALS was initiated in August 2018. According to, the randomized, placebo-controlled study includes 231 participants and will aim to evaluate the efficacy and safety of the agent in patients with ALS by focusing on primary outcome measures, such as the Combined Assessment of Function and Survival (CAFS), over a 76-week stretch.

Other secondary outcome measures included in the study are time to permanent assisted ventilation (PAV) or tracheostomy or death, change from baseline to Week 76 on the revised ALS Functional Rating Scale (ALSFRS-R), and change from baseline to Week 76 in Slow Vital Capacity (SVC).

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A previous phase 2/3 clinical trial (NCT00706147) showed that arimoclomol was safe, and that patients tolerated it well. The trial enrolled 36 patients with superoxide dismutase 1 (SOD1)-related ALS and were given either arimoclomol 200 mg TID or placebo 3 times a day for up to 12 months.

Results showed that after adjusting for riluzole exposure and baseline ALSFRS-R score, survival favored arimoclomol with a hazard ratio (HR) of 0.77 (95% CI, 0.32—1.80). ALSFRS-R score and predicted forced expiratory volume in 6 seconds (FEV6) declined more slowly in the arimoclomol group, with treatment differences of 0.5 points per month (95% CI, –0.63 to 1.63) and 1.24% predicted FEV6 per month (95% CI, ­–2.77 to 5.25), respectively. Combined Assessment of Function and Survival similarly favored arimoclomol.2

As Stratton mentyioned, in June 2016, the drug received fast track designation for the treatment of Niemann-Pick disease type C 3 weeks after the FDA had given it a positive review of the AIDNPC investigational new drug (IND) application.3 Three years later, in December 2019, the drug was granted fast track designation for the treatment of sporadic Inclusion Body Myositis (sIBM). Results from the phase 2/3 trial with arimoclomol for sIBM are expected in the first half of 2021.4


1. Orphazyme’s arimoclomol receives US fast track designation in Amyotrophic Lateral Sclerosis [news release]. Copenhagen, Denmark: Orphazyme. Published May 22, 2020. Accessed June 1, 2020.

2. Benatar M, Wuu J, Anderson PM, et al. Randomized, double blind, placebo-controlled trial of arimoclomol in rapidly progressive SOD1 ALS. Neurology. 2018 Feb 13;90 (7):e565—e574. doi: 10.1212/WNL.0000000000004960

3. FDA grants fast track designation for the phase 3 clinical investigation of arimoclomol as a treatment of Niemann-pick disease type c [news release]. Copenhagen, Denmark: Orphazyme. Published June 27, 2016. Accessed June 1, 2020.

4. Orphazyme’s arimoclomol receives US fast track designation in sporadic Inclusion Body Myositis [news release]. Copenhagen, Denmark. Published December 18, 2019. Accessed June 1, 2020.

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