An investigational medication for amyotrophic lateral sclerosis (ALS), a deadly neurological ailment that may cause paralysis, has been given the green light in Canada. This opens the door to a new treatment option for ALS, a condition for which there are very few viable treatments.
The approval is contingent upon the pharmaceutical firm subsequently providing further proof demonstrating that the therapy is effective. It is likely to be of major interest to patients with A.L.S. (amyotrophic lateral sclerosis) in the United States, where the same therapy — AMX0035, which is going to be marketed as Albrioza — is being evaluated by the Food and Drug Administration, which has raised questions about the effectiveness of the treatment. Currently, the FDA is evaluating the therapy.
Earlier this year, the Food and Drug Administration conducted a review that concluded Albrioza was safe to use, but that there was insufficient evidence to suggest that it was effective in either helping patients live longer or slowing the rate at which they lost functions such as the ability to control their muscles, speak, or breathe without assistance. In March, a committee comprised of independent experts to the FDA reached the conclusion, by a razor-thin margin, that the treatment was not yet ready to be approved.
The Food and Drug Administration (FDA) was going to make its final judgement this month, but they announced not too long ago that they would be extending the deadline to September 29 because they needed more time to study further analyses of data that were provided by the firm.
A.L.S., commonly known as Lou Gehrig’s disease, is diagnosed in around 6,000 individuals throughout the globe every year and often results in death within two to five years after the first diagnosis. In the United States, there are only two drugs that have been licenced to treat A.L.S.: riluzole, which may prolong life by several months, and edaravone, which can reduce development by around 33 percent. Both of these treatments are administered intravenously.
The medicine Albrioza is a mixture of two medications that are already on the market. It comes in the form of a powder that has a bitter flavour and is taken twice a day, either by mixing it with water and drinking it or by ingesting it via a feeding tube. Amylyx Pharmaceuticals, a tiny firm based in Massachusetts, is the one responsible for its production. The company’s founders, Justin Klee and Joshua Cohen, had the idea for the treatment back when they were undergraduate students at Brown University a little over a decade ago.
They hypothesised that by combining taurursodiol, a supplement that is occasionally used to regulate liver enzymes, and sodium phenylbutyrate, a medication for a paediatric urea disorder, they would be able to protect neurons by preventing the dysfunction of two structures within cells: the mitochondria and the endoplasmic reticulum. In other words, they would be able to protect neurons by ensuring that the mitochondria and the endoplasmic reticulum
In the Phase 2 trial, two-thirds of the 137 participants were given Albrioza, and over the course of 24 weeks, they experienced a decline in physical abilities that was 25 percent slower than the decline experienced by the participants who were given a placebo. This translated to a difference of 2.32 points on a scale that rates 12 different physical abilities, including walking, speaking, swallowing, dressing, handwriting, and breathing.
The open-label extension trial included 90 of those patients, 34 of whom had been assigned to the placebo group. These patients started receiving the medicine around seven months after those who had gotten it from the very beginning. According to the findings published by Amylyx, patients who were given the medication for the greatest period of time had a median of around 6.5 months extra time before being hospitalised, being placed on a ventilator, or passing away. More data, which revealed an extra advantage, was released by researchers who participated in the trial only last month.
The majority of the funding for Amylyx’s research on Albrioza came from the company itself; however, the A.L.S. Association also made a contribution of $2.2 million utilising funds that were donated in response to the 2014 Ice Bucket Challenge. Amylyx has committed to using the proceeds from the sale of the medicine to return one hundred fifty percent of the association’s award in order to finance more research.
Patients in the clinical studies were required to have experienced symptoms during the previous 18 months before to the study and to have been impacted in at least three body locations, both of which are indicators of a disease that is advancing quickly. Although the approval from Health Canada did not impose any limitations on which A.L.S. patients could take Albrioza, the founders of Amylyx and Dr. Genge both stated that it is possible that such limitations could be established by the Canadian coverage system or by pharmaceutical formularies in some of Canada’s provinces.