Courier treatment of "gradual freezing syndrome", innovative treatments are eligible for priority review

▎The content team editor of WuXi AppTec recently announced that Amylyx Pharmaceuticals announced that the US FDA has accepted the New Drug Application (NDA) of AMX0035 for the treatment of amyotrophic lateral sclerosis (ALS) and granted priority review status
.

The PDUFA target date is set at June 29, 2022
.

AMX0035 is a compound preparation of two drugs, sodium phenylbutrate and taurursodiol
.

They can improve the health of mitochondria and endoplasmic reticulum in cells, thereby delaying the death of nerve cells
.

Preclinical trials have shown that the synergistic effect of the combination of these two drugs can reduce the death of nerve cells due to oxidative stress by 90%
.

 Amylyx will also submit an Expanded Access Program application to the FDA for use in the next few months for patients who cannot participate in Phase 3 clinical trials of the drug
.

The NDA submitted to the US FDA is based on positive data obtained from a phase 2 clinical trial involving 137 ALS patients
.

The trial reached its primary efficacy endpoint, that is, according to the revised ALS function rating scale, at the end of the 6-month randomization phase, the decline in motor function of ALS patients treated with AMX0035 was significantly slowed
.

In addition, a 3-year follow-up of all randomized subjects showed that patients who were treated with AMX0035 at the beginning of the trial had a risk of death compared with patients who were initially treated with placebo and switched to AMX0035 during the open-label phase.
A reduction of 44% (HR 0.
56; 95% CI, 0.
34-0.
92)
.

And the median survival time of the group who always received AMX0035 treatment was 6.
5 months longer than that of the group who initially received placebo (25.
0 months vs.
18.
5 months)
.

In terms of safety, overall, within 24 weeks, the adverse events and discontinuation rates reported between the AMX0035 and placebo groups were basically similar, but the incidence of gastrointestinal events in the AMX0035 group was higher
.

Relevant detailed data has been published in the "New England Journal of Medicine" and Muscle and Nerve
.

Dr.
Merit Cudkowicz, a professor of neurology at Harvard Medical School and co-lead investigator of the trial, said: "With the acceptance of AMX0035's NDA, we believe we are one step closer to (bringing) potential new treatment options
.

We look forward to seeing progress in benefiting patients through the regulatory review process as we continue to study its therapeutic potential in the global phase 3 clinical trial of AMX0035
.

"Reference: [1] Amylyx Pharmaceuticals Announces FDA Acceptance and Priority Review of New Drug Application (NDA) for AMX0035 for the Treatment of ALS.
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