Approved Drugs for ALS |
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There is no cure for ALS, and so far no drugs have been proven to stop or reverse the progression of the disease. However, there are now three FDA-approved drugs that can help slow progression, one that can help treat bulbar symptoms, and one that targets the SOD1 gene.
Qalsody (tofersen) approved by FDA
In April 2023, the U.S. Food and Drug Administration (FDA) granted accelerated approval for the first treatment to target a genetic cause of ALS. “Qalsody targets the SOD1 gene,” explains Dr. Terry Heiman-Patterson, “which, when mutated, produces a protein that is toxic to the nerve cells.” According to the FDA, approximately 2% of ALS cases are associated with mutations in the SOD1 gene.
Qalsody received conditional approval based on early clinical trial data that suggested a likely benefit. Continued approval may be contingent on the results of an ongoing phase 3 trial. Learn more
Qalsody received conditional approval based on early clinical trial data that suggested a likely benefit. Continued approval may be contingent on the results of an ongoing phase 3 trial. Learn more
Rilutek® (riluzole)
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Studies have shown that riluzole, approved by the U.S. Food and Drug Administration (FDA) in 1995, can extend the life of ALS patients for a few months, particularly if you begin taking it shortly after diagnosis.
Side effects are rare, but can include dizziness, nausea, weakness, drowsiness, stomach pain, and vomiting. Some people cannot tolerate it. Medicare, Medicaid, the VA, and many private insurance policies cover it. |
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If you decide to take riluzole, discuss with your ALS clinic team. For the first few months, you may need to have blood drawn to monitor your liver function enzymes.
If you have trouble swallowing pills, the FDA approved a liquid form of riluzole called Tiglutik in 2018.
If you have trouble swallowing pills, the FDA approved a liquid form of riluzole called Tiglutik in 2018.
Radicava® (edaravone)
In 2017, the FDA approved Radicava to treat ALS after one six-month clinical trial in Japan in which the drug maker claimed that Radicava slowed the decline of physical function by 33%.
Since Radicava’s release, there have been no other clinical trials. Some studies and reviews have highlighted its benefits, though others have questioned its effectiveness. Benefits may be more likely if you begin early in your disease progression.
Radicava was administered through intravenous infusions (IVs) until the oral formulation, Radicava ORS, was approved by the FDA in May 2022. The FDA writes: “Radicava ORS is self-administered and can be taken at home. After fasting overnight, Radicava ORS should be taken in the morning orally or through a feeding tube. The oral medication has the same dosing regimen as Radicava—an initial treatment cycle of daily dosing for 14 days, followed by a 14-day drug-free period and subsequent treatment cycles consisting of daily dosing for 10 out of 14-day periods, followed by 14-day drug-free periods.”
Regarding side effects, the FDA writes: “The most common side effects of Radicava are bruising (contusions), problems walking (gait disturbances), and headaches. Fatigue is also a possible side effect from Radicava ORS. Radicava and Radicava ORS can have serious side effects associated with allergic reactions including hives, rash, and shortness of breath. For patients with sulfite sensitivity, sodium bisulfite—an ingredient in Radicava and Radicava ORS—could cause a type of allergic reaction that can be life-threatening.”
Radicava can be expensive. It is typically covered under Medicare Part B. Coverage through private insurance varies.
The Radicava website addresses many common questions. Ask your neurologist, ALS clinic team, and local ALS organization for their thoughts on whether it could help you.
Since Radicava’s release, there have been no other clinical trials. Some studies and reviews have highlighted its benefits, though others have questioned its effectiveness. Benefits may be more likely if you begin early in your disease progression.
Radicava was administered through intravenous infusions (IVs) until the oral formulation, Radicava ORS, was approved by the FDA in May 2022. The FDA writes: “Radicava ORS is self-administered and can be taken at home. After fasting overnight, Radicava ORS should be taken in the morning orally or through a feeding tube. The oral medication has the same dosing regimen as Radicava—an initial treatment cycle of daily dosing for 14 days, followed by a 14-day drug-free period and subsequent treatment cycles consisting of daily dosing for 10 out of 14-day periods, followed by 14-day drug-free periods.”
Regarding side effects, the FDA writes: “The most common side effects of Radicava are bruising (contusions), problems walking (gait disturbances), and headaches. Fatigue is also a possible side effect from Radicava ORS. Radicava and Radicava ORS can have serious side effects associated with allergic reactions including hives, rash, and shortness of breath. For patients with sulfite sensitivity, sodium bisulfite—an ingredient in Radicava and Radicava ORS—could cause a type of allergic reaction that can be life-threatening.”
Radicava can be expensive. It is typically covered under Medicare Part B. Coverage through private insurance varies.
The Radicava website addresses many common questions. Ask your neurologist, ALS clinic team, and local ALS organization for their thoughts on whether it could help you.
RELYVRIO (AMX0035)
In September 2022, the U.S. Food and Drug Administration approved RELYVRIO (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.”
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 about ten months. The drug is not a cure, does not restore function, and does not stop decline.
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.
RELYVRIO was approved after 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. Though some scientists were concerned by the lack of convincing clinical trial data, the ALS community was overwhelmingly enthusiastic about the approval of RELYVRIO. Read more
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.”
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 about ten months. The drug is not a cure, does not restore function, and does not stop decline.
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.
RELYVRIO was approved after 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. Though some scientists were concerned by the lack of convincing clinical trial data, the ALS community was overwhelmingly enthusiastic about the approval of RELYVRIO. Read more
Nuedexta®
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For ALS patients who experience pseudobulbar affect (PBA), which is characterized by sudden and unpredictable episodes of laughing or crying, Nuedexta is an FDA-approved oral medication that can help regulate emotional control.
Patients take two capsules per day and side effects are rare. A secondary benefit is that Nuedexta may improve speech and swallowing for some patients whose bulbar muscles have been affected. |
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Other treatments for ALS
Prescription drugs are not the only way people living with ALS can address symptoms, have a better quality of life, and potentially live longer.
- Studies have shown that attending an ALS clinic regularly can help extend your life by one year or more, in addition to improving your quality of life.
- Respiratory equipment, such as a bilevel machine, may also help you live longer. When breathing becomes more difficult, some people choose to extend their lives by getting a tracheotomy and going on full-time mechanical ventilation, though this requires 24/7 care.
- Getting a feeding tube can help you maintain your weight, have more energy, and get adequate nutrition and hydration.
- Some people living with ALS choose to explore non-pharmaceutical treatments such as supplements. Some alternative treatments have received positive clinical trial results.
