Options mapped
Amyotrophic lateral sclerosis (ALS): options by country
Sourced options by country plus visit-prep questions for Amyotrophic lateral sclerosis (ALS). Each line links to its regulator, HTA, or guideline source. This page maps options; it does not recommend or rank them.
Options mappedRare diseaseLast checked June 2026
What this page does
Maps options by country
It maps sourced options by country alongside diagnosis wording, stage, test results, specialists, and trial-search terms.
What it does not do
Does not choose treatment
It does not rank treatments, recommend a choice, or decide clinical fit.
Where it comes from
Built on trusted sources
Every option links to a trusted regulator, HTA, or guideline source, and the list grows as new sources pass verification.
Information to gather before the next visit
- Has genetic testing evaluated SOD1 and other familial ALS genes?
- Which currently marketed ALS drugs are available through the treating center or insurer?
- Is any proposed therapy approved, accelerated-approved, withdrawn, or trial-only in the United States?
- Is the pathway EU authorization, national reimbursement, or NHS access?
Trial-search terms to discuss
Options by country
Treatments by country
Regulatory and access status by country, from official sources. It shows what exists and where — not a recommendation.
United States
- Riluzole / Rilutek and generics; edaravone / Radicava and Radicava ORS; tofersen / Qalsody; sodium phenylbutyrate plus taurursodiol / Relyvrio noted as withdrawn/discontinued for new U.S. patients[1]FDA-approvedSOD1 mutation for tofersen; no biomarker restriction captured for riluzole or edaravone; Adult ALS for edaravone; ALS generally for riluzole; SOD1 mutation-associated ALS for tofersen. · Tofersen is accelerated approval and continued approval is contingent on confirmatory evidence. Relyvrio is recorded as withdrawn/discontinued for new U.S. patients, so it is not cataloged as a current treatment option except as an access-history caveat. Confidence/conflicts: High for current U.S. riluzole, edaravone, tofersen, and Relyvrio withdrawal/discontinuation status. No source conflict identified. Riluzole (Rilutek/generics) and edaravone (Radicava/Radicava ORS) are FDA-approved, marketed ALS standard-of-care disease-modifying drugs. Tofersen (Qalsody) is under FDA accelerated approval for SOD1-mutation ALS; continued approval is contingent on the ongoing ATLAS confirmatory trial. ONLY Relyvrio (sodium phenylbutyrate + taurursodiol / AMX0035) was withdrawn — it failed the Phase 3 PHOENIX trial and FDA withdrew its NDA approval effective 29 Aug 2025; it is not a current option except as access history. The 'did not show benefit' framing applies to Relyvrio alone, never to riluzole/edaravone/tofersen.
- Riluzole / Rilutek and generics; edaravone / Radicava and Radicava ORS; tofersen / Qalsody; sodium phenylbutyrate plus taurursodiol / Relyvrio noted as withdrawn/discontinued for new U.S. patients[1]FDA-approvedSOD1 mutation for tofersen; no biomarker restriction captured for riluzole or edaravone; Adult ALS for edaravone; ALS generally for riluzole; SOD1 mutation-associated ALS for tofersen. · Tofersen is accelerated approval and continued approval is contingent on confirmatory evidence. Relyvrio is recorded as withdrawn/discontinued for new U.S. patients, so it is not cataloged as a current treatment option except as an access-history caveat. Confidence/conflicts: High for current U.S. riluzole, edaravone, tofersen, and Relyvrio withdrawal/discontinuation status. No source conflict identified. Riluzole (Rilutek/generics) and edaravone (Radicava/Radicava ORS) are FDA-approved, marketed ALS standard-of-care disease-modifying drugs. Tofersen (Qalsody) is under FDA accelerated approval for SOD1-mutation ALS; continued approval is contingent on the ongoing ATLAS confirmatory trial. ONLY Relyvrio (sodium phenylbutyrate + taurursodiol / AMX0035) was withdrawn — it failed the Phase 3 PHOENIX trial and FDA withdrew its NDA approval effective 29 Aug 2025; it is not a current option except as access history. The 'did not show benefit' framing applies to Relyvrio alone, never to riluzole/edaravone/tofersen.
- Riluzole / Rilutek and generics; edaravone / Radicava and Radicava ORS; tofersen / Qalsody; sodium phenylbutyrate plus taurursodiol / Relyvrio noted as withdrawn/discontinued for new U.S. patients[1]FDA-approvedSOD1 mutation for tofersen; no biomarker restriction captured for riluzole or edaravone; Adult ALS for edaravone; ALS generally for riluzole; SOD1 mutation-associated ALS for tofersen. · Tofersen is accelerated approval and continued approval is contingent on confirmatory evidence. Relyvrio is recorded as withdrawn/discontinued for new U.S. patients, so it is not cataloged as a current treatment option except as an access-history caveat. Confidence/conflicts: High for current U.S. riluzole, edaravone, tofersen, and Relyvrio withdrawal/discontinuation status. No source conflict identified. Riluzole (Rilutek/generics) and edaravone (Radicava/Radicava ORS) are FDA-approved, marketed ALS standard-of-care disease-modifying drugs. Tofersen (Qalsody) is under FDA accelerated approval for SOD1-mutation ALS; continued approval is contingent on the ongoing ATLAS confirmatory trial. ONLY Relyvrio (sodium phenylbutyrate + taurursodiol / AMX0035) was withdrawn — it failed the Phase 3 PHOENIX trial and FDA withdrew its NDA approval effective 29 Aug 2025; it is not a current option except as access history. The 'did not show benefit' framing applies to Relyvrio alone, never to riluzole/edaravone/tofersen.
European Union
- Riluzole / Rilutek; tofersen / Qalsody; supportive care including physical, occupational or speech therapy and breathing support as described by EMA[2]EMA authorisedSOD1 mutation for Qalsody/tofersen; ALS form of MND for riluzole; adult SOD1 mutation-associated ALS for Qalsody/tofersen; symptom and breathing support alongside disease-modifying options. · EMA notes EU-wide authorization and member-state price/reimbursement decisions are separate. NICE TA20 is older but remains an official appraisal for riluzole; it does not establish tofersen NHS reimbursement. Confidence/conflicts: High for EU Qalsody and UK riluzole claims; UK tofersen reimbursement remains source-pending.
- Riluzole / Rilutek; tofersen / Qalsody; supportive care including physical, occupational or speech therapy and breathing support as described by EMA[2]EMA authorisedSOD1 mutation for Qalsody/tofersen; ALS form of MND for riluzole; adult SOD1 mutation-associated ALS for Qalsody/tofersen; symptom and breathing support alongside disease-modifying options. · EMA notes EU-wide authorization and member-state price/reimbursement decisions are separate. NICE TA20 is older but remains an official appraisal for riluzole; it does not establish tofersen NHS reimbursement. Confidence/conflicts: High for EU Qalsody and UK riluzole claims; UK tofersen reimbursement remains source-pending.
- Riluzole / Rilutek; tofersen / Qalsody; supportive care including physical, occupational or speech therapy and breathing support as described by EMA[2]EMA authorisedSOD1 mutation for Qalsody/tofersen; ALS form of MND for riluzole; adult SOD1 mutation-associated ALS for Qalsody/tofersen; symptom and breathing support alongside disease-modifying options. · EMA notes EU-wide authorization and member-state price/reimbursement decisions are separate. NICE TA20 is older but remains an official appraisal for riluzole; it does not establish tofersen NHS reimbursement. Confidence/conflicts: High for EU Qalsody and UK riluzole claims; UK tofersen reimbursement remains source-pending.
Japan
- Tofersen / Qalsody; riluzole; edaravone; mecobalamin; symptomatic medications such as opioids for respiratory distress/suffering and antispasmodics for spasm as described in PMDA review discussion[3]PMDA-approved (Japan)SOD1 mutation for tofersen; SOD1 mutation-associated ALS for tofersen; ALS treatment context for riluzole, edaravone, and mecobalamin. · PMDA English translation is reference material and Japanese original takes precedence. PMDA notes patient selection should be by physicians with adequate knowledge and experience in ALS treatment, with benefit-risk assessment for individual patients. Confidence/conflicts: High for Japan tofersen review and PMDA-stated local ALS treatment landscape; no conflict identified. Availability/reimbursement outside the approving regulator not established.
- Tofersen / Qalsody; riluzole; edaravone; mecobalamin; symptomatic medications such as opioids for respiratory distress/suffering and antispasmodics for spasm as described in PMDA review discussion[3]PMDA-approved (Japan)SOD1 mutation for tofersen; SOD1 mutation-associated ALS for tofersen; ALS treatment context for riluzole, edaravone, and mecobalamin. · PMDA English translation is reference material and Japanese original takes precedence. PMDA notes patient selection should be by physicians with adequate knowledge and experience in ALS treatment, with benefit-risk assessment for individual patients. Confidence/conflicts: High for Japan tofersen review and PMDA-stated local ALS treatment landscape; no conflict identified. Availability/reimbursement outside the approving regulator not established.
- Tofersen / Qalsody; riluzole; edaravone; mecobalamin; symptomatic medications such as opioids for respiratory distress/suffering and antispasmodics for spasm as described in PMDA review discussion[3]PMDA-approved (Japan)SOD1 mutation for tofersen; SOD1 mutation-associated ALS for tofersen; ALS treatment context for riluzole, edaravone, and mecobalamin. · PMDA English translation is reference material and Japanese original takes precedence. PMDA notes patient selection should be by physicians with adequate knowledge and experience in ALS treatment, with benefit-risk assessment for individual patients. Confidence/conflicts: High for Japan tofersen review and PMDA-stated local ALS treatment landscape; no conflict identified. Availability/reimbursement outside the approving regulator not established.
Australia
- riluzole (Rilutek)[4]ApprovedALS treatment in Australia, as stated by the Australian product information. · The product information includes hepatic, pregnancy/lactation, and pediatric caveats; this entry records label status only, not PBS subsidy or individual suitability. Confidence/conflicts: High for Australian riluzole label status; no conflict identified. Availability/reimbursement outside the approving regulator not established.
- riluzole (Rilutek)[4]ApprovedALS treatment in Australia, as stated by the Australian product information. · The product information includes hepatic, pregnancy/lactation, and pediatric caveats; this entry records label status only, not PBS subsidy or individual suitability. Confidence/conflicts: High for Australian riluzole label status; no conflict identified. Availability/reimbursement outside the approving regulator not established.
- tofersen (Qalsody)[5]TGA-registered (Australia)mutation/defect in the superoxide dismutase 1 (SOD1) gene; adult SOD1-associated ALS in Australia. · The approval is provisional. The TGA source describes SOD1-ALS as a rare ALS form and records regulatory status, not payer access, genetic-testing access, or individual suitability. Confidence/conflicts: High for Australian provisional-approval status; no conflict identified. Availability/reimbursement outside the approving regulator not established.
- tofersen (Qalsody)[5]TGA-registered (Australia)mutation/defect in the superoxide dismutase 1 (SOD1) gene; adult SOD1-associated ALS in Australia. · The approval is provisional. The TGA source describes SOD1-ALS as a rare ALS form and records regulatory status, not payer access, genetic-testing access, or individual suitability. Confidence/conflicts: High for Australian provisional-approval status; no conflict identified. Availability/reimbursement outside the approving regulator not established.
Canada
- riluzole (Mylan-Riluzole and riluzole class context)[6]ApprovedCanadian ALS treatment context for riluzole. · The monograph explicitly states riluzole is not a cure and that symptomatic management remains important. This entry records product-monograph context, not a personalized recommendation or payer status. Confidence/conflicts: Medium-high for Canadian riluzole monograph context; current brand-specific DPD status should be checked for payer implementation. Availability/reimbursement outside the approving regulator not established.
- riluzole (Mylan-Riluzole and riluzole class context)[6]ApprovedCanadian ALS treatment context for riluzole. · The monograph explicitly states riluzole is not a cure and that symptomatic management remains important. This entry records product-monograph context, not a personalized recommendation or payer status. Confidence/conflicts: Medium-high for Canadian riluzole monograph context; current brand-specific DPD status should be checked for payer implementation. Availability/reimbursement outside the approving regulator not established.
- tofersen (Qalsody)[7]Health Canada approvedmutation in the superoxide dismutase 1 (SOD1) gene; adult SOD1-associated ALS in Canada. · The product monograph states clinical benefit remains to be confirmed and continued approval may depend on confirmatory trials. Health Canada's RDS states the pivotal trial did not adequately demonstrate benefit on the primary endpoint but showed promising evidence and a favourable benefit-risk assessment for a serious condition. Confidence/conflicts: High for conditional Canadian authorization; uncertainty is explicitly recorded in the Health Canada sources. Availability/reimbursement outside the approving regulator not established.
- tofersen (Qalsody)[7]Health Canada approvedmutation in the superoxide dismutase 1 (SOD1) gene; adult SOD1-associated ALS in Canada. · The product monograph states clinical benefit remains to be confirmed and continued approval may depend on confirmatory trials. Health Canada's RDS states the pivotal trial did not adequately demonstrate benefit on the primary endpoint but showed promising evidence and a favourable benefit-risk assessment for a serious condition. Confidence/conflicts: High for conditional Canadian authorization; uncertainty is explicitly recorded in the Health Canada sources. Availability/reimbursement outside the approving regulator not established.
- tofersen (Qalsody), with background-care context including riluzole and edaravone in CADTH material[8]Health Canada approvedmutation in the superoxide dismutase 1 (SOD1) gene; Canadian reimbursement-with-conditions context for adult SOD1-associated ALS. · CADTH is a reimbursement body, not a prescribing label. It states clinical benefit is of undetermined clinical significance and depends on conditions, including genetic confirmation and jurisdictional implementation. Confidence/conflicts: Medium-high for CADTH reimbursement context; provincial implementation and genetic-testing access remain variable.
- tofersen (Qalsody), with background-care context including riluzole and edaravone in CADTH material[8]Health Canada approvedmutation in the superoxide dismutase 1 (SOD1) gene; Canadian reimbursement-with-conditions context for adult SOD1-associated ALS. · CADTH is a reimbursement body, not a prescribing label. It states clinical benefit is of undetermined clinical significance and depends on conditions, including genetic confirmation and jurisdictional implementation. Confidence/conflicts: Medium-high for CADTH reimbursement context; provincial implementation and genetic-testing access remain variable.
- tofersen (Qalsody), with background-care context including riluzole and edaravone in CADTH material[8]Health Canada approvedmutation in the superoxide dismutase 1 (SOD1) gene; Canadian reimbursement-with-conditions context for adult SOD1-associated ALS. · CADTH is a reimbursement body, not a prescribing label. It states clinical benefit is of undetermined clinical significance and depends on conditions, including genetic confirmation and jurisdictional implementation. Confidence/conflicts: Medium-high for CADTH reimbursement context; provincial implementation and genetic-testing access remain variable.
Sources
- DailyMed / U.S. National Library of Medicine — official drug label · official drug label
- European Medicines Agency (EMA) — regulator EPAR · regulator EPAR
- Pharmaceuticals and Medical Devices Agency / MHLW — regulator review and deliberation report · regulator review and deliberation report
- Australian medicine-information repository / Rilutek product information — official product information · official product information
- Therapeutic Goods Administration (TGA) — Australian Prescription Medicine Decision Summary · Australian Prescription Medicine Decision Summary
- Health Canada / Mylan product monograph repository — official product monograph · official product monograph
- Biogen Canada / Health Canada product monograph format — official product monograph with conditions · official product monograph with conditions
- CADTH via NCBI Bookshelf — reimbursement recommendation / health technology review · reimbursement recommendation / health technology review
This is official regulatory and access status only — not medical advice, not a recommendation, and not a statement about eligibility. Whether any option fits depends on your situation and your oncology team. Status changes over time; confirm the current position with the linked source. Last checked 2026-06-12.
Beyond approved care
In clinical trials & emerging options
Options that are not — or not yet — an approved standard where you live: studies, clinical trials, off-label use, and early evidence that your own oncologist may not raise. Each is labeled by how strong the evidence is. A listing here is information to research and discuss with your team; it does not mean a treatment is proven, safe for you, or available today.
In clinical trials
- Tofersen / BIIB067; ALN-SOD; VHB937; masitinib plus riluzole; personalized or standard repetitive transcranial magnetic stimulation (rTMS) plus exercise; music therapyClinical trial · NCT02623699Clinical trialTrial only (NCT02623699)Korea · SOD1 mutation for tofersen and ALN-SOD trials; no biomarker restriction captured for VHB937, masitinib, rTMS, or music therapy records; SOD1 mutation ALS for tofersen and ALN-SOD records; early-stage ALS for VHB937; ALS patients on stable riluzole in masitinib trial; motor neuron disease in Thailand rTMS/exercise study; bulbar/respiratory supportive music therapy in Russia/UK record. · Trial status varies from recruiting to completed, active-not-recruiting, and unknown. Some records require stable riluzole/edaravone or specific respiratory function thresholds and do not imply eligibility for any individual. Confidence/conflicts: High for registry-listed trial/supportive cells; no national approval claim is made from these records. ClinicalTrials.gov — clinical-trial registry
- Tofersen / BIIB067; ALN-SOD; VHB937; masitinib plus riluzole; personalized or standard repetitive transcranial magnetic stimulation (rTMS) plus exercise; music therapyClinical trial · NCT02623699Clinical trialTrial only (NCT02623699)Korea · SOD1 mutation for tofersen and ALN-SOD trials; no biomarker restriction captured for VHB937, masitinib, rTMS, or music therapy records; SOD1 mutation ALS for tofersen and ALN-SOD records; early-stage ALS for VHB937; ALS patients on stable riluzole in masitinib trial; motor neuron disease in Thailand rTMS/exercise study; bulbar/respiratory supportive music therapy in Russia/UK record. · Trial status varies from recruiting to completed, active-not-recruiting, and unknown. Some records require stable riluzole/edaravone or specific respiratory function thresholds and do not imply eligibility for any individual. Confidence/conflicts: High for registry-listed trial/supportive cells; no national approval claim is made from these records. ClinicalTrials.gov — clinical-trial registry
- Tofersen / BIIB067; ALN-SOD; VHB937; masitinib plus riluzole; personalized or standard repetitive transcranial magnetic stimulation (rTMS) plus exercise; music therapyClinical trial · NCT02623699Clinical trialTrial only (NCT02623699)Korea · SOD1 mutation for tofersen and ALN-SOD trials; no biomarker restriction captured for VHB937, masitinib, rTMS, or music therapy records; SOD1 mutation ALS for tofersen and ALN-SOD records; early-stage ALS for VHB937; ALS patients on stable riluzole in masitinib trial; motor neuron disease in Thailand rTMS/exercise study; bulbar/respiratory supportive music therapy in Russia/UK record. · Trial status varies from recruiting to completed, active-not-recruiting, and unknown. Some records require stable riluzole/edaravone or specific respiratory function thresholds and do not imply eligibility for any individual. Confidence/conflicts: High for registry-listed trial/supportive cells; no national approval claim is made from these records. ClinicalTrials.gov — clinical-trial registry
- Tofersen / BIIB067; ALN-SOD; VHB937; masitinib plus riluzole; personalized or standard repetitive transcranial magnetic stimulation (rTMS) plus exercise; music therapyClinical trial · NCT02623699Clinical trialTrial only (NCT02623699)Korea · SOD1 mutation for tofersen and ALN-SOD trials; no biomarker restriction captured for VHB937, masitinib, rTMS, or music therapy records; SOD1 mutation ALS for tofersen and ALN-SOD records; early-stage ALS for VHB937; ALS patients on stable riluzole in masitinib trial; motor neuron disease in Thailand rTMS/exercise study; bulbar/respiratory supportive music therapy in Russia/UK record. · Trial status varies from recruiting to completed, active-not-recruiting, and unknown. Some records require stable riluzole/edaravone or specific respiratory function thresholds and do not imply eligibility for any individual. Confidence/conflicts: High for registry-listed trial/supportive cells; no national approval claim is made from these records. ClinicalTrials.gov — clinical-trial registry
- Edaravone / Radicava; NTF001 intrathecal gene therapy; SNUG01 intrathecal therapyClinical trialClinical trialReported in a clinical trialChina · ALS treatment for edaravone; adult ALS early-phase/investigator-initiated intrathecal gene or experimental therapy contexts for NTF001 and SNUG01. · Edaravone approval source is a company announcement rather than a directly fetched NMPA label. NTF001 and SNUG01 remain trial-framed and have active-not-recruiting/not-yet-recruiting statuses in fetched records. Confidence/conflicts: Medium-high for China edaravone approval because source is manufacturer announcement naming NMPA; high for registry trial cells. Primary NMPA label remains an active gap. Mitsubishi Tanabe Pharma Corporation — manufacturer regulatory announcement
- Edaravone / Radicava; NTF001 intrathecal gene therapy; SNUG01 intrathecal therapyClinical trialClinical trialReported in a clinical trialChina · ALS treatment for edaravone; adult ALS early-phase/investigator-initiated intrathecal gene or experimental therapy contexts for NTF001 and SNUG01. · Edaravone approval source is a company announcement rather than a directly fetched NMPA label. NTF001 and SNUG01 remain trial-framed and have active-not-recruiting/not-yet-recruiting statuses in fetched records. Confidence/conflicts: Medium-high for China edaravone approval because source is manufacturer announcement naming NMPA; high for registry trial cells. Primary NMPA label remains an active gap. Mitsubishi Tanabe Pharma Corporation — manufacturer regulatory announcement
- Edaravone / Radicava; NTF001 intrathecal gene therapy; SNUG01 intrathecal therapyClinical trialClinical trialReported in a clinical trialChina · ALS treatment for edaravone; adult ALS early-phase/investigator-initiated intrathecal gene or experimental therapy contexts for NTF001 and SNUG01. · Edaravone approval source is a company announcement rather than a directly fetched NMPA label. NTF001 and SNUG01 remain trial-framed and have active-not-recruiting/not-yet-recruiting statuses in fetched records. Confidence/conflicts: Medium-high for China edaravone approval because source is manufacturer announcement naming NMPA; high for registry trial cells. Primary NMPA label remains an active gap. Mitsubishi Tanabe Pharma Corporation — manufacturer regulatory announcement
A clinical-trial listing or early report shows an option is being studied — not that it works, that it is safe for any one person, or that a site is enrolling today. Whether any of these fits is a conversation for your oncology team and the trial team. Last checked 2026-06-12.