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CompilationFan Dongdong
A few days ago, the US Food and Drug Administration (FDA) has accepted Amylyx amyotrophic lateral sclerosis (ALS, gradual freezing) AMX0035 to submit a new drug application (NDA)
.
After recent discussions with the FDA, Amylyx announced the NDA submission on Wednesday morning
.
Just in April of this year, the FDA stated to Amylyx that before submitting an NDA application, Amylyx needs to supplement additional placebo-controlled study data
In 2017, AMX0035 was awarded the title of rare disease drug by the FDA
.
The drug is composed of sodium phenylbutyrate and taurine diol
In a 2:1 placebo-controlled trial of 137 participants, half of the patients received AMX0035 for 24 weeks
.
The results of the trial showed that patients in the AMX0035 treatment group had a survival benefit of up to 6.
Justin Klee, founder and co-CEO of Amylyx, said, “AMX0035 is the first therapy
that has shown clinical benefits in terms of function and survival in the development of this progressively fatal disease gradual cryosurgery.
Klee pointed out that the company will Continue to follow up the follow-up results of the trial survival analysis
Like many brain diseases, gradual freezing is still a difficult target for drug developers, and the FDA has stricter supervision in this area
.
At the end of last year, a New York biotechnology company named Brainstorm Cell Therapeutics disclosed that its stem cell-based gradual freezing therapy did not meet the main goal of a later trial
In addition to actively developing the US market, Amylyx is also actively promoting regulatory breakthroughs in other countries
.
In June of this year, the company submitted a New Drug Application (NDS) to Health Canada, and it is expected that it will be approved within 10 months
Reference source:
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