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Canada has ALS . An experimental therapy is approved for (amyotrophic lateral sclerosis), a fatal and paralyzing neurological disorder, that adds a new treatment option to a disease for which there are few effective treatments, according to an article published in new York Times,
Approval, first in the world for treatment, AMX0035,—which will be marketed in Canada as Albrioza—is made on condition that the drug company provides more evidence that the treatment works. This is likely to be of great interest to ALS patients in the United States, where the same therapy is being evaluated by the Food and Drug Administration (FDA), which has raised questions about the efficacy of the treatment.
article of new York Times show that the review The FDA earlier this year found the therapy safe, but said there wasn’t enough evidence that it was effective in helping patients live longer or slowing the rate at which they lose muscle control, Lose functions such as speaking and hearing. without help. A committee of independent FDA advisors voted narrowly in March that the therapy was not ready for approval.
The US regulatory agency was scheduled to issue a decision later this month, but eventually extended the deadline to September 29, asking it to review further analysis of the data submitted by the company and Time is required.
Meanwhile, Kalneet Balas, president and CEO of the ALS Association, one of the patient advocacy organizations pushing for FDA approval, said, “We hope that Americans living with ALS will try to reach Albrioza in Canada, as will the We hear people are trying. Buy it on Amazon”.
two drugs in america
ALS, also known as Lou Gehring’s disease, It is diagnosed in more than 6,000 people worldwide each year and often results in death within two to five years., There are only two approved drugs for ALS in the US: riluzole, which can increase survival by several months, and edaravone, which can slow progression by about 33%.
Albrioza is a combination of two existing compounds in the form of a bitter-tasting powder that is mixed with water and drunk or swallowed through a feeding tube twice a day. It is manufactured by Amylix Pharmaceuticals, a small Massachusetts company.Whose founders, Justin Clay and Joshua Cohen, conceived of medicine less than a decade ago as students at Brown University.
His idea was that torsodiol, a supplement sometimes used to regulate liver enzymes, and sodium phenylbutyrate, a drug for a pediatric urea disorder, could protect neurons by preventing dysfunction of two structures in cells. May: mitochondria and endoplasmic reticulum.
Data comparing Albrioza to placebo so far comes from Phase II trials (which is smaller than Phase 3 studies, which are usually required by regulatory agencies); Additional information comes from an open-label extension study that followed some patients after the trial ended, while they were intentionally taking the drug. The FDA typically requires two clinical trials of the drug for approval, But in cases of severe disease with few available treatments, you may want to consider clinical trial evidence and additional supporting data.
The Authority of Canada (Health Canada) approved Albrioza under a formula called Notice of Compliance with ConditionsWhich allows for the approval of drugs that show promise for serious diseases, but have incomplete evidence that they work. The central condition established by the agency is that Amylyx “verify clinical benefit of this drug” with data from an ongoing Phase 3 clinical trial. and is expected to be completed in 2024, according to agency documents sent to the company on Friday. The company should also conduct additional pharmacological studies and provide safety reports from time to time. In addition, the agency’s communication warns that “patients should be informed”.
Health Canada said in a statement: “Canadians living with ALS have limited options for their treatment. Following a thorough review of the information provided by the company in the drug submission, Health Canada concluded that the benefits of Albrioza outweigh the risks when used as intended.”
The FDA has a similar program called accelerated approval that allows conditional approval of drugs with incomplete evidence of effectiveness, but that program also requires that a compound be recognized as part of the biological mechanism underlying a disease. be shown to target., Experts have said that if Albrioza does not receive standard FDA approval, it is unlikely to meet the criteria for accelerated approval because little is known about the underlying biology of ALS and how Albrioza might address it.
Last month, 38 doctors treating ALS patients sent a letter to the FDA for approval. The ALS Association said its campaign for approval generated more than 6,000 emails in recent weeks asking the agency to flag the drug.
Caleb Alexander, a member of the FDA’s independent advisory committee, who voted in March that there was not enough evidence that the therapy worked, said he still thinks the FDA should wait for the results of the Phase 3 trial. and “it would be a mistake to pass it on the basis of trial alone.”
Dr Alexander, an internist and epidemiologist at the Johns Hopkins Bloomberg School of Public Health, said there was a dire need for effective treatments for ALS, but for Albrioza, “it is unfortunate, but the magnitude of the need not met is not known.” Consistent with the quality of evidence to date.”
He added that “approval in Canada can only add to the pressure the FDA is facing to rule favorably and approve this product.”
It is generally illegal for Americans to import drugs that have not been approved for personal use in the US. But the FDA website lists some exceptions. This may apply to Albrioza, even though the drug has no serious safety concerns and is intended to treat “a serious condition for which no effective treatment is available in the US”.
Director of the ALS Association Global Center for Excellence at the Montreal Neurological Institute, Dr. Angela Geng, who received a fee from Amylix to serve on an advisory board, said US patients could legally receive Albrioza in Canada if prescribed by a Canadian physician and obtained from a Canadian pharmacy. from, although they will not be covered for insurance coverage under Canada’s public or private system.
In an interview, Cohen and Clay declined to disclose the price Amylex is considering for Albrioza, saying it is still being negotiated. He said Medicare would be available to people paying privately in about six weeks, but it would take longer, possibly months, for people to receive coverage under Canada’s public system. He said Amylex has already provided Albrioza free of charge to 250 patients in the US under a compassionate use agreement.
As of last summer, the FDA recommended that Amylyx not apply for approval until the drug had completed its phase 3 trial.But in July, officials began suggesting that Amylix file an application for approval using existing data. Pressure from ALS lobbyists came after the approval of the new Alzheimer’s drug, EduHelm, was highly controversial, with many experts saying there was not enough data to show EduHelm worked.
Phase 2 Testing
In the Phase 2 trial, two-thirds of 137 participants who received Albrioza experienced a 25% slower decline at 24 weeks than participants who received placebo: a 2.32-point less decline on a 48-point scale. Which assesses 12 physical skills including walking, talking, swallowing, dressing, handwriting and breathing.
The open-label extension study included 90 of those patients, including 34 from the placebo group, who started taking the drug approximately seven months later than those who received it from the beginning. Amylex reported that those who received the longest treatment had an average of about 6.5 months before they were hospitalized, put on a ventilator, or died. Researchers involved in the study published more data last month that suggested additional benefits.
Amylyx funded most of its Albrioza research, but the ALS Association contributed $2.2 million using money raised through the 2014 Ice Bucket Challenge. Amylyx agreed to use the sale of the drug to pay 150% of the association’s grant to fund further research.
Clinical trials included patients who developed symptoms within 18 months prior to testing and affected at least three areas of the body, usually with symptoms of rapidly progressing disease. Health Canada’s approval did not impose any restrictions on which ALS patients could take Albrioza, but the founders of Amylex and Dr. Genge said such limits may be set by the Canadian coverage system or by pharmaceutical formulary in some Canadian provinces. , the article ends. new York Times.
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