ALS Drug Relyvrio Removed from the Market
Due to disappointing results from the Phase 3 clinical trial, Amylyx Pharmaceuticals announced on April 4, 2024 that it has removed AMX0035 (Relyvrio/Albrioza) from the market in the U.S. and Canada. Patients currently taking the drug may continue using it for free in consultation with their physician. Learn more
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On Sept. 29, 2022 the U.S. Food and Drug Administration approved AMX0035 for the treatment of ALS. Clinical trial results from 2020 indicated that the drug 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.” |
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After Rilutek (riluzole) and Radicava (edaravone), AMX0035 becomes just the third FDA-approved medication prescribed for slowing the progression of ALS. AMX0035 will be marketed in the United States under the name RELYVRIO.
What is RELYVRIO (AMX0035)?
Relyvrio is a powder that combines two existing drugs (sodium phenylbutyrate and taurursodiol) that are commonly used and have been proven safe in prior studies. Relyvrio appears to slow the progression of ALS and potentially extend life by a median of ten months. The drug is not a cure and does not restore function.
The lead researcher said that trial results were modest but meaningful. She believes that Relyvrio will become one of a handful of medications patients take to slow the progression of ALS until there is a cure.
The lead researcher said that trial results were modest but meaningful. She believes that Relyvrio will become one of a handful of medications patients take to slow the progression of ALS until there is a cure.
How do you take Relyvrio?
You just dissolve one packet of Relyvrio powder into eight ounces of room temperature water and take twice daily, either orally or through a feeding tube. For the first three weeks, you take one packet a day. Relyvrio should be taken before a snack or a meal. You can take this drug on its own or along with other ALS medications.
What are the side effects of Relyvrio?
Researchers and the FDA believe that Relyvrio doesn’t pose serious safety concerns. The most common side effects in the clinical trial were diarrhea, abdominal pain, nausea, and upper respiratory tract infection.
The FDA notes that “Relyvrio contains taurursodiol, a bile acid, which may cause worsening diarrhea in patients with disorders that interfere with bile acid circulation. These patients should consider consulting with a specialist before taking Relyvrio. The prescribing information includes additional information on risks associated with Relyvrio.”
You can learn more on Amylyx Pharmaceuticals’ patient information page.
The FDA notes that “Relyvrio contains taurursodiol, a bile acid, which may cause worsening diarrhea in patients with disorders that interfere with bile acid circulation. These patients should consider consulting with a specialist before taking Relyvrio. The prescribing information includes additional information on risks associated with Relyvrio.”
You can learn more on Amylyx Pharmaceuticals’ patient information page.
How much will Relyvrio cost?
The morning after the drug’s approval, Amylyx set the listing price around $158,000 for a year’s supply—about $12,000 less than Radicava. How much you actually pay will depend on your insurance coverage and the final negotiated price. Amylyx says it plans to provide the drug “at no cost to U.S. patients that are uninsured or underinsured, under certain conditions.”
The Amylyx Care Team (ACT) Support Program can help you navigate insurance, overcome potential barriers to access, and provide financial assistance for those with out-of-pocket costs. People living with ALS, caregivers, and healthcare professionals in the U.S. can call 1-866-318-2989 or email [email protected] to ask questions and get direct support.
The Amylyx Care Team (ACT) Support Program can help you navigate insurance, overcome potential barriers to access, and provide financial assistance for those with out-of-pocket costs. People living with ALS, caregivers, and healthcare professionals in the U.S. can call 1-866-318-2989 or email [email protected] to ask questions and get direct support.
What are ALS experts saying about Relyvrio?
Though some scientists have been concerned by the lack of convincing clinical trial data, the ALS community has been overwhelmingly enthusiastic about the approval of Relyvrio.
The ALS Association, Answer ALS Foundation, I AM ALS, Les Turner ALS Foundation, and Team Gleason said in a joint statement, “With today’s approval, we are encouraged that RELYVRIO can offer people living with ALS and their families the potential of more time with functional independence… This is significant for people living with ALS, their loved ones, caregivers, clinicians, researchers, and advocacy, as we now have a new treatment option that could be a big step forward for the future of ALS care.”
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 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.”
The ALS Association, Answer ALS Foundation, I AM ALS, Les Turner ALS Foundation, and Team Gleason said in a joint statement, “With today’s approval, we are encouraged that RELYVRIO can offer people living with ALS and their families the potential of more time with functional independence… This is significant for people living with ALS, their loved ones, caregivers, clinicians, researchers, and advocacy, as we now have a new treatment option that could be a big step forward for the future of ALS care.”
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 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.”
Why was it approved before getting phase 3 clinical trial data?
Clinical trial results published on September 3, 2020 indicated that AMX0035 could slow functional decline in a meaningful way and extend life for people living with ALS.
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.
During the application process, 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.
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.
During the application process, 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.
