Express delivery gives patients more time to spend with their families, and the innovative treatment of "gradual freezing syndrome" is one step closer to being approved

▎The content team editor of WuXi AppTec recently announced that Amylyx Pharmaceuticals has submitted a New Drug Application (NDA) for AMX0035 to the US FDA for the treatment of amyotrophic lateral sclerosis (ALS)
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AMX0035 is a compound preparation of two drugs, sodium phenylbutrate and taurursodiol
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They can improve the health of mitochondria and endoplasmic reticulum in cells, thereby delaying the death of nerve cells
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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%
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This innovative therapy originated from the thinking of two university students, Mr.
Joshua Cohen and Mr.
Justin Klee, in the university dormitory
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This NDA submission is based on the positive data obtained from a phase 2 clinical trial.
The trial reached its primary efficacy endpoint, which was measured according to the revised ALS functional rating scale and received AMX0035 treatment at the end of the 6-month randomization phase.
The decline of the patient's motor function was significantly slowed down
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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)
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In terms of safety, overall, during the 24-week randomization period, 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
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Relevant detailed data has been published in the "New England Journal of Medicine" and Muscle and Nerve
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Amylyx also plans to submit a marketing authorization application (MAA) for AMX0035 to the European Medicines Agency (EMA) at about the end of 2021, and to initiate a global phase 3 clinical trial in the fourth quarter of 2021, with about 600 subjects To evaluate the safety and efficacy of AMX0035
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Dr.
Sabrina Paganoni, the principal investigator of the Phase 2 clinical trial, said: “For ALS patients and related medical staff, this is a major advancement and provides hope for potential new treatment options
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This therapy has been proven to slow ALS.
As the disease progresses, give patients more time to spend with their families
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"Reference: [1] Amylyx Pharmaceuticals Submits New Drug Application (NDA) for AMX0035 for the Treatment of ALS.
Retrieved November 2, 2021, from https://www .
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com/news/home/20211102005320/en/Disclaimer: WuXi AppTec's content team focuses on introducing global biomedical health research progress
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This article is for the purpose of information exchange only.
The opinions expressed in the article do not represent the position of WuXi AppTec, nor does it mean that WuXi AppTec supports or opposes the views in the article
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This article is not a treatment recommendation either
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If you need guidance on treatment plans, please go to a regular hospital for treatment
.