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▎Editor of WuXi AppTec content team
Relyvrio is a combination of two drugs, sodium phenylbutyrate and taurursodiol
.
They can improve the health of intracellular mitochondria and endoplasmic reticulum, 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%.
The FDA has granted Relyvrio orphan drug qualification and priority review qualification
.
.
According to WuXi AppTec's internal database, as of the end of May this year, nearly 100 ALS drug candidates have entered the clinical development stage
.
I look forward to the smooth development of more innovative therapies and the early arrival of
patients.
To learn more about FDA's approved new drugs, please click on the following image to access our Mini Program
Today, the U.
S.
FDA announced the approval of Relyvrio, an oral fixed-dose formulation of sodium phenybutyrate and taurate, for the treatment of amyotrophic lateral sclerosis (ALS
).
The process of getting this treatment has been a lot of twists and turns
.
In March, an FDA advisory committee voted 6:4 that clinical data do not yet support the efficacy
of the therapy.
Subsequently, Amylyx submitted further analysis of the clinical trial data as well as supporting data from other clinical trials
.
AMX0035 is also approved for a conditional listing
by Canadian regulators for the first time.
At the second FDA Advisory Board in September this year, committee members voted 7:2 in favor of FDA approval of
the therapy.
Today this therapy finally successfully crossed the finish line!
Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease that affects nerve cells in the brain and spinal cord, also known as "frostbite
".
The motor neurons of the brain and spine of patients with this disease will continue to die, resulting in muscle weakness and paralysis, from inability to walk, inability to speak, swallow, and breathe
.
The average life expectancy of ALS patients after diagnosis is only four years
.
The exact pathogenesis
of the disease is unclear.
Relyvrio is a combination of two drugs, sodium phenylbutyrate and taurursodiol
.
They can improve the health of intracellular mitochondria and endoplasmic reticulum, 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%.
The FDA has granted Relyvrio orphan drug qualification and priority review qualification
.
The approval is based on positive data from a Phase 2 clinical trial that enrolled 137 patients with ALS
.
The trial reached its primary efficacy endpoint, which was a significant slowdown
in motor function in ALS patients receiving Relyvrio at the end of the 6-month randomization phase, as measured by the revised ALS Functional Rating Scale.
In addition, follow-up of all randomized subjects for up to 3 years showed a 44% reduction in the risk of death (HR 0.
56; 95% CI, 0.
34-0.
92)
compared with patients who initially received placebo and switched to Relyvrio at the open-label stage.
And the group that always received Relyvrio had a median survival of 6.
5 months longer than the group of patients who initially received placebo (25.
0 months vs.
18.
5 months).
Safety, overall, the reported adverse events and discontinuation rates were substantially similar between the Relyvrio and placebo groups over a 24-week period, but the incidence of gastrointestinal events was higher
in the Relyvrio group.
"Amylyx's goal is to enable every patient who qualifies for Relyvrio to use the drug in the quickest and most efficient way because we know that ALS patients and their families have no time to wait any longer," said Joshua Cohen and Mr.
Justin Klee, Co-CEO of Amylyx
.
”
.
According to WuXi AppTec's internal database, as of the end of May this year, nearly 100 ALS drug candidates have entered the clinical development stage
.
I look forward to the smooth development of more innovative therapies and the early arrival of
patients.
To learn more about FDA's approved new drugs, please click on the following image to access our Mini Program
WuXi AppTec provides integrated, end-to-end new drug development and manufacturing services to the global biopharmaceutical industry, covering the fields
of chemical drug development and production, biological research, preclinical testing and clinical trial development, cell and gene therapy development, testing and production.
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.
