Update: On September 29, 2022 the FDA approved AMX0035, a new ALS drug that will be marketed in the United States under the name RELYVRIO. This webpage is now outdated. Please visit our new RELYVRIO page for current information.
|
Clinical trial results published on September 3, 2020 indicated that a new ALS drug, AMX0035, could slow functional decline in a meaningful way and extend life for people living with ALS.
The ALS Association, which supported the development of AMX0035, wrote that it “could mean the difference between a person with ALS being able to feed themselves versus being fed, or the difference between needing a wheelchair versus not needing one.” |
|
The encouraging results, however, came from a phase 2 clinical trial. Typically, a more lengthy phase 3 trial is needed to confirm the effectiveness and safety of a new drug before it can be made available to the public.
In March 2022, an FDA advisory committee narrowly rejected the drug due to lack of data proving its effectiveness. Yet strong pushback from ALS advocacy groups such as I AM ALS and the ALS Association persuaded the FDA to take the unusual step of reconvening the committee to reevaluate the drug in light of a new analysis by drugmaker Amylyx Pharmaceuticals.
In their Sept. 7, 2022 meeting, the advisory committee voted 7-2 in support of approving AMX0035. And then on Sept. 29, 2022, the FDA granted final approval of AMX0035, which will be marketed under the name Relyvrio. Canada granted conditional approval for this same drug in June 2022.
In March 2022, an FDA advisory committee narrowly rejected the drug due to lack of data proving its effectiveness. Yet strong pushback from ALS advocacy groups such as I AM ALS and the ALS Association persuaded the FDA to take the unusual step of reconvening the committee to reevaluate the drug in light of a new analysis by drugmaker Amylyx Pharmaceuticals.
In their Sept. 7, 2022 meeting, the advisory committee voted 7-2 in support of approving AMX0035. And then on Sept. 29, 2022, the FDA granted final approval of AMX0035, which will be marketed under the name Relyvrio. Canada granted conditional approval for this same drug in June 2022.
What is AMX0035?
Relyvrio is a powder that is dissolved in water and taken twice a day either orally or through a feeding tube. It combines two existing drugs (sodium phenylbutyrate and taurursodiol), both of which have been found safe in prior studies. Researchers believe that Relyvrio is safe. Some trial participants experienced mild side effects.
Relyvrio is not a cure, does not restore function, and does not stop decline. It appears to slow the progression of ALS and potentially extend life by ten months. The lead researcher said that the trial results were modest but meaningful. She believes that Relyvrio will become one of a handful of medications patients will take to slow the progression of ALS until there is a cure.
Relyvrio is not a cure, does not restore function, and does not stop decline. It appears to slow the progression of ALS and potentially extend life by ten months. The lead researcher said that the trial results were modest but meaningful. She believes that Relyvrio will become one of a handful of medications patients will take to slow the progression of ALS until there is a cure.
What are ALS experts saying?
In May 2022, a few dozen prominent ALS clinicians sent a letter to the FDA urging the approval of AMX0035. They wrote, “We find sufficient evidence that AMX0035 is safe, well-tolerated, and provides a clinically meaningful benefit. As clinicians, we ask FDA to grant approval so we can work with our patients to determine if AMX0035 is right for them.”
Citing no substantive safety concerns and the potential for benefit, they implored the FDA to exercise flexibility and approve the drug before results of the current phase 3 trial become available in late 2023 or early 2024. “People living with ALS,” they noted, “have stated their willingness repeatedly to accept significant risk both of safety and uncertainty of benefit.”
“Under these circumstances,” they concluded, “and in the case of a rapidly progressing and fatal disease, we can ethically prescribe this treatment, and should be allowed to offer this option to our patients.”
Amylyx stated that if AMX0035 were granted approval and then its phase 3 trial results showed it to be ineffective, it would pull the drug from the market.
Citing no substantive safety concerns and the potential for benefit, they implored the FDA to exercise flexibility and approve the drug before results of the current phase 3 trial become available in late 2023 or early 2024. “People living with ALS,” they noted, “have stated their willingness repeatedly to accept significant risk both of safety and uncertainty of benefit.”
“Under these circumstances,” they concluded, “and in the case of a rapidly progressing and fatal disease, we can ethically prescribe this treatment, and should be allowed to offer this option to our patients.”
Amylyx stated that if AMX0035 were granted approval and then its phase 3 trial results showed it to be ineffective, it would pull the drug from the market.
