The United States has approved the marketing of Relyvrio, a medicine for the treatment of patients with amyotrophic lateral sclerosis (ALS), an incurable disease, as announced by the Food and Drug Administration (FDA, for its acronym in English).
The pharmaceutical regulator has given the drug its go-ahead despite doubts about its efficacy without waiting for the outcome of a larger clinical trial, given the lack of alternatives.
Trials with Relyvrio have been funded in part by funds raised from the
ice bucket challenge,
which went viral a few years ago.
The FDA states that the efficacy of Relyvrio (sodium phenylbutyrate/taurursodiol) for the treatment of ALS was demonstrated in a 24-week, randomized, double-blind, placebo-controlled, parallel-group, multicenter study.
In the trial, 137 adult ALS patients were randomized to receive the drug or placebo.
Patients treated with Relyvrio experienced a slower rate of decline in a clinical assessment of their functionality compared to those who received placebo.
Additionally, a longer overall survival was observed in a long-term analysis of patients who initially received the compound versus those who received placebo.
The documentation published by the FDA, however, admits that a regulatory flexibility has been applied in this case, since it recognizes that in the results there is "a degree of residual uncertainty about the evidence of effectiveness that exceeds" what is usual for the approval of drugs. medicines.
"However, given the serious and life-threatening nature of ALS and the significant unmet need, this level of uncertainty is acceptable at this stage and consideration of these results in the context of regulatory flexibility is appropriate."
sick pressure
ALS is a rare disease that attacks and kills the nerve cells that control voluntary muscles.
Voluntary muscles produce movements such as chewing, walking, breathing, and speaking.
ALS causes nerves to lose the ability to activate specific muscles, causing the muscles to weaken and leading to paralysis.
It is a progressive disease that continues to worsen over time.
Death from respiratory failure occurs in most cases, usually within three to five years from the appearance of the first symptoms.
In the absence of alternatives, doctors and patient associations insistently advocated the approval of the drug, developed by Amylyx Pharmaceuticals, a laboratory based in Cambridge (Massachsetts).
Amylyx promised to withdraw it from the market if it finally turned out to be ineffective.
The company made a similar commitment to the Canadian regulator, which approved the drug in June.
The FDA prioritized the review and also granted it orphan drug designation, which provides incentives to help and encourage the development of drugs for rare diseases.
Amylyx is a company founded in 2013 by Josh Coen and Justin Kleem valued at around $1.8 billion.
When they were students at Brown University (in Providence, Rhode Island).
Both conceived the idea of combining sodium phenylbutyrate, a drug indicated in urea cycle disorders, with taurursodiol to protect brain neurons in degenerative diseases such as ALS.
Cohen and Kleen have overseen the drug's development "from its inception as a dorm room concept to its current status," the company says on its website.
“FDA approval of Relyvrio is an exciting milestone for the ALS community and is an important step toward achieving our mission to one day end suffering from neurodegenerative diseases,” said Cohen and Klee. it's a statement.
“We want to sincerely thank the wider ALS community, including healthcare professionals and ALS sufferers, for their guidance, support of our clinical programs and for sharing their experiences with us.
Their stories have inspired us and helped our team better understand the ALS clock, instilling in us a deep sense of urgency that will keep us going.
This is just the beginning and much remains to be done”, they added.
The company has not yet announced a price for the drug in Canada or the United States, but it does state its goal of making it accessible to all who need it by providing financial assistance options.
Amylyx shares soared on the stock market as early as this Thursday in off-market operations.
The company has called a conference with analysts and investors for this Friday.
The FDA says that the benefits outweigh the risk and that the "drug appears to be well tolerated with no significant safety signals of concern."
The most common adverse reactions it causes are diarrhoea, abdominal pain, nausea and upper respiratory tract infection, according to the drug's package insert.
Relyvrio contains taurursodiol, a bile acid, which may cause worsening of diarrhea in patients with disorders that interfere with the circulation of bile acids.
“This approval provides another important treatment option for ALS, a life-threatening disease that currently has no cure,” Billy Dunn, MD, director of the Office of Neuroscience at the Center for Drug Evaluation and Research at the University of California, said in a statement. FDA.
"The FDA remains committed to facilitating the development of more treatments for ALS," he added.
About 5,000 people are diagnosed with ALS each year in the United States, and about 20,000 Americans currently have the disease, according to the FDA.
Amylyx raises the figure to 29,000.
In Spain there are between 3,000 and 4,000 ALS patients.
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