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    Explainer

    ALS treatments guide: riluzole, edaravone, tofersen and what's next

    Three drugs approved in the US slow ALS modestly or target one genetic form: riluzole, edaravone and tofersen. A fourth, Relyvrio, was withdrawn in 2024. None stops or reverses the disease.[1][2][3] The evidence varies. Riluzole probably adds two to three months of median survival, edaravone's benefit was shown in a selected subgroup, and tofersen was approved on a biomarker after missing its main clinical endpoint.[4][5][6][7]

    Editor reviewedStrict sourcingUpdated ExplainerHealth and medicine, Life sciences, Neuroscience and brain-computer interfaces

    This guide compares the medicines used for ALS, using published trial results and regulators’ findings. None of them stops or reverses the disease.[1] We do no testing or clinical work of our own. This page is not medical advice and gives no dosing guidance. Decisions about treatment should be made with a neurologist or ALS care team. Any figures below are from the named study and apply to the population it enrolled.

    At a glance

    TreatmentWho it is forKey evidenceStatus (Oct 2026)
    Riluzole (Rilutek; liquid and dissolving forms)Adults with ALSCochrane review of randomised trials: probably about 2-3 months longer median survival[4]Approved[8]
    Edaravone (Radicava, IV or oral)Adults with ALSJapanese phase 3 RCT in a selected early-stage subgroup: slower functional decline over 24 weeks[9]Approved[10]
    Tofersen (Qalsody, spinal injection)SOD1-ALS onlyPhase 3 RCT missed its functional endpoint but lowered SOD1 and neurofilament[7]FDA accelerated approval 2023; EU 2024[6][11]
    Sodium phenylbutyrate/taurursodiol (Relyvrio)Was for adults with ALSLarger trial failed to confirm benefit[3]Withdrawn 2024[3]
    Ulefnersen (investigational)FUS-ALSCompany-reported phase 3 topline: primary endpoint met[12]Not approved[13]

    Riluzole

    Riluzole is a tablet, also available as a thickened liquid or a tablet that dissolves on the tongue. It is thought to protect motor neurons by lowering glutamate, a chemical messenger. Trials showed it may extend survival by a few months.[8] The NHS describes it as a medicine that can help slow the progression of ALS.[8]

    A 2012 Cochrane review pooled randomised trials with 974 riluzole-treated and 503 placebo-treated patients. It concluded the drug is reasonably safe and probably prolongs median survival by about two to three months.[4] In the pooled analysis, one-year survival was 58% versus 49%, and median survival rose from 11.8 to 14.8 months. Raised liver enzymes were more common with riluzole.[14] This is randomised-trial evidence, the strongest type, but the effect is small.

    Edaravone

    Edaravone is an antioxidant, given through a drip or by mouth. It has been shown to slow loss of function in some people with ALS.[10] Its trial included only people early in the disease who met strict criteria.[5]

    An earlier phase 3 study found no significant difference on the ALSFRS-R functional scale. A second trial enrolled only the subgroup that looked responsive in a post-hoc analysis.[5] In that 24-week Japanese RCT of 137 randomised patients, ALSFRS-R fell by 5.01 points with IV edaravone versus 7.50 with placebo.[9] The authors themselves describe efficacy in “a small subset” of people with ALS, so the results may not apply to everyone with the disease.[5]

    Tofersen for SOD1-ALS

    Tofersen is a genetic medicine for the roughly 2% of people whose ALS is caused by a faulty SOD1 gene.[15] It is an antisense drug that lowers production of the harmful SOD1 protein. It is given by spinal injection.[16] It is approved only for ALS linked to a SOD1 mutation.[6] For how antisense drugs work, see how RNA silencing drugs work.

    In the phase 3 VALOR trial of 108 adults, tofersen reduced CSF SOD1 and plasma neurofilament light over 28 weeks but did not significantly improve clinical endpoints.[7] In the open-label extension, people who started early declined by 6.0 ALSFRS-R points at 52 weeks, against 9.5 for those who started later. Neurologic serious adverse events occurred in 7% of recipients.[17] The FDA granted accelerated approval in April 2023, based on the drop in neurofilament light, a biomarker reasonably likely to predict benefit.[6] The EU authorised it under exceptional circumstances in May 2024.[11] EMA lists serious side effects including myelitis and aseptic meningitis.[18] The confirmatory ATLAS trial in presymptomatic carriers expects primary completion in 2027.[19]

    Withdrawn and investigational drugs

    Relyvrio was approved in September 2022 on the strength of one smaller trial. A larger trial did not confirm the benefit, and the manufacturer withdrew it in 2024.[3] Ulefnersen, an antisense drug for FUS-ALS, met its primary endpoint in a phase 3 trial, according to a September 2026 company announcement.[12] These are topline results. Full data have not yet been peer-reviewed, and the drug is not approved.[13]

    Care beyond drugs

    Drugs are one part of care. Multidisciplinary teams provide physical, occupational and speech therapy.[20] Noninvasive ventilation, feeding tubes and communication aids such as voice banking matter more as ALS advances.[21][22] The NHS says people with MND should see their care team every 2 or 3 months.[20] For new results, see the ALS research tracker.

    Next in the crash course

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    Developing in Health

    Questions readers ask

    What is the best treatment for ALS?

    There is no single best treatment; choices depend on the person and should be made with an ALS care team. No drug stops or reverses ALS, and NINDS describes the effects of disease-modifying drugs as modest.[1][2]

    How much does riluzole help?

    A 2012 Cochrane review concluded riluzole probably prolongs median survival by about two to three months; in the pooled trials one-year survival was 58% with riluzole versus 49% with placebo.[4][14]

    Who can get tofersen?

    Tofersen (Qalsody) is approved only for ALS linked to a mutation in the SOD1 gene, about 2% of cases, and is given by spinal injection.[6][15][16]

    Why was Relyvrio withdrawn?

    It was approved in September 2022 based on one smaller trial. A larger trial failed to confirm the results, and the manufacturer removed it from the market in 2024.[3]

    Is ulefnersen available?

    Not as of October 2026. Its phase 3 trial in FUS-ALS met its primary endpoint in September 2026, but it has not been approved by any regulator.[12][13]

    Sources

    · 22 claims from 10 publishers

    Each numbered claim is a statement we checked against the sources listed with it. Tap a number in the text to see its sources in place.

    1. [1]

      There is no treatment that stops or reverses ALS; approved drugs may prolong survival, slow decline or manage symptoms. confirmedas of 2026-10-11

    2. [2]

      NINDS says the FDA has approved one symptom-managing and four disease-modifying ALS therapies, but the impact of the disease-modifying therapies is modest. confirmedas of 2026-10-11

    3. [3]

      The FDA approved sodium phenylbutyrate/taurursodiol (Relyvrio) in September 2022 based on a single smaller trial; a larger trial failed to confirm the results and the manufacturer withdrew it from the market in 2024. confirmedas of 2026-10-11

    4. [4]

      A 2012 Cochrane review of randomised trials (974 riluzole-treated and 503 placebo-treated patients) concluded riluzole is reasonably safe and probably prolongs median survival by about two to three months. confirmedas of 2012-03-14

    5. [5]

      The edaravone trial authors said the drug showed efficacy in a small subset of people with ALS, after an earlier phase 3 study had found no significant difference in the full population. confirmedas of 2017-05-15

    6. [6]

      The FDA granted accelerated approval to Qalsody (tofersen) for ALS associated with SOD1 mutations in April 2023, based on a reduction in plasma neurofilament light, a blood marker of nerve injury. confirmedas of 2023-04-25

    7. [7]

      In the phase 3 VALOR trial (108 adults with SOD1-ALS), tofersen lowered SOD1 in spinal fluid and neurofilament light in plasma over 28 weeks but did not significantly improve the primary functional endpoint. confirmedas of 2022-09-22

    8. [8]

      Riluzole (Rilutek) is an oral drug thought to reduce motor neuron damage by lowering glutamate; trials showed it may extend survival by a few months, and liquid and dissolving forms exist for people with swallowing problems. confirmedas of 2026-10-11

    9. [9]

      In a 24-week Japanese phase 3 trial of 137 randomised patients with early-stage ALS chosen by criteria from an earlier post-hoc analysis, functional scores (ALSFRS-R) fell by 5.01 points with intravenous edaravone versus 7.50 with placebo. confirmedas of 2017-05-15

    10. [10]

      Edaravone (Radicava) is an antioxidant given intravenously or orally that has been shown to slow functional decline in some people with ALS; Radicava ORS can be taken by mouth or through a feeding tube. confirmedas of 2026-10-11

    11. [11]

      Qalsody received an EU marketing authorisation under exceptional circumstances on 29 May 2024, with a requirement to provide long-term data and study people who do not yet have symptoms. confirmedas of 2024-06-14

    12. [12]

      On 22 September 2026 Otsuka and Ionis announced that the phase 3 FUSION trial of ulefnersen in FUS-ALS met its primary endpoint, a joint-rank measure of function and survival through day 505 (p=0.0005 versus placebo). confirmedas of 2026-09-22

    13. [13]

      As of October 2026 ulefnersen is not approved anywhere; Otsuka and Ionis plan to discuss expedited submission with the FDA and to present detailed results at a medical congress and in a peer-reviewed journal. confirmedas of 2026-09-22

    14. [14]

      In the Cochrane analysis, one-year survival was 58% with riluzole versus 49% with placebo, median survival rose from 11.8 to 14.8 months, and raised liver enzymes were more frequent with riluzole. confirmedas of 2012-03-14

    15. [15]

      Citing CDC, the FDA said in 2023 that between 16,000 and 32,000 Americans are living with ALS, and that about 2% of cases (fewer than 500 US patients) are associated with SOD1 mutations. confirmedas of 2023-04-25

    16. [16]

      Tofersen is an antisense oligonucleotide that targets SOD1 messenger RNA to reduce production of SOD1 protein, and it is given by spinal (intrathecal) injection. confirmedas of 2023-04-25

    17. [17]

      At 52 weeks in VALOR's open-label extension, functional scores fell by 6.0 points in participants who started tofersen early versus 9.5 in those who started 28 weeks later; neurologic serious adverse events occurred in 7% of tofersen recipients. confirmedas of 2022-09-22

    18. [18]

      Common tofersen side effects include pain, fatigue and raised white cells in spinal fluid; EMA lists serious side effects including myelitis, raised pressure around the brain and aseptic meningitis. confirmedas of 2024-06-14

    19. [19]

      The ATLAS confirmatory trial is testing tofersen in 158 presymptomatic adult SOD1 mutation carriers with elevated neurofilament; as of October 2026 it is active but not recruiting, with primary completion estimated for August 2027. confirmedas of 2026-10-11

    20. [20]

      Care for ALS is best provided by multidisciplinary teams, including physical, occupational and speech therapy; the NHS says people with MND should see their care team every 2 or 3 months. confirmedas of 2026-10-11

    21. [21]

      As breathing muscles weaken, noninvasive ventilation through a mask may help, first at night and later full time, and a feeding tube can reduce the risk of choking and pneumonia when eating becomes difficult. confirmedas of 2026-10-11

    22. [22]

      Speech therapists help people with ALS keep communicating, including eye-tracking speech synthesizers and voice banking, which stores a person's own voice for later use. confirmedas of 2026-10-11

    Revision history (1)
    1. Page created.

    Created Oct 11, 2026. Last reviewed by an editor on Oct 11, 2026. Next scheduled review: Jan 11, 2027.

    Cite this page

    "ALS treatments guide: riluzole, edaravone, tofersen and what's next." ContentLora, updated Oct 11, 2026. https://contentlora.com/explain/als-treatments-guide

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