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Gaucher disease: options by country

Sourced options by country plus visit-prep questions for Gaucher disease. 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

  • Which Gaucher subtype and manifestations are being treated: systemic type 1, non-CNS type 3, or neurologic disease?
  • Is enzyme replacement therapy, substrate reduction therapy, or a switch between therapies being discussed?
  • Are infusion reactions, CYP2D6 status, liver disease, drug interactions, and venous access relevant to the option?
  • Is the option being considered under EMA authorization, NICE access, or a local national reimbursement route?

Trial-search terms to discuss

Gaucher disease clinical trial

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

  • Imiglucerase (Cerezyme); velaglucerase alfa (VPRIV); taliglucerase alfa (Elelyso); eliglustat (Cerdelga); miglustat (Zavesca)[1]FDA-approvedGBA1 / acid beta-glucosidase deficiency; CYP2D6 metabolizer status for eliglustat; Long-term enzyme replacement or substrate-reduction therapy as defined in FDA labels; Zavesca specifically when ERT is not a therapeutic option. · Cerezyme, VPRIV, and Elelyso labels include hypersensitivity/anaphylaxis precautions and administration under qualified supervision. Cerdelga requires CYP2D6 metabolizer assessment and has liver/drug-interaction restrictions. Zavesca is not framed as first-line ERT replacement in the label. Confidence/conflicts: High for U.S. label indications. No comparative efficacy or availability beyond FDA labeling is inferred.
  • Imiglucerase (Cerezyme); velaglucerase alfa (VPRIV); taliglucerase alfa (Elelyso); eliglustat (Cerdelga); miglustat (Zavesca)[1]FDA-approvedGBA1 / acid beta-glucosidase deficiency; CYP2D6 metabolizer status for eliglustat; Long-term enzyme replacement or substrate-reduction therapy as defined in FDA labels; Zavesca specifically when ERT is not a therapeutic option. · Cerezyme, VPRIV, and Elelyso labels include hypersensitivity/anaphylaxis precautions and administration under qualified supervision. Cerdelga requires CYP2D6 metabolizer assessment and has liver/drug-interaction restrictions. Zavesca is not framed as first-line ERT replacement in the label. Confidence/conflicts: High for U.S. label indications. No comparative efficacy or availability beyond FDA labeling is inferred.
  • Imiglucerase (Cerezyme); velaglucerase alfa (VPRIV); taliglucerase alfa (Elelyso); eliglustat (Cerdelga); miglustat (Zavesca)[1]FDA-approvedGBA1 / acid beta-glucosidase deficiency; CYP2D6 metabolizer status for eliglustat; Long-term enzyme replacement or substrate-reduction therapy as defined in FDA labels; Zavesca specifically when ERT is not a therapeutic option. · Cerezyme, VPRIV, and Elelyso labels include hypersensitivity/anaphylaxis precautions and administration under qualified supervision. Cerdelga requires CYP2D6 metabolizer assessment and has liver/drug-interaction restrictions. Zavesca is not framed as first-line ERT replacement in the label. Confidence/conflicts: High for U.S. label indications. No comparative efficacy or availability beyond FDA labeling is inferred.

European Union

  • Imiglucerase (Cerezyme); eliglustat (Cerdelga); miglustat (Zavesca); enzyme replacement therapy including imiglucerase or velaglucerase alfa[2]EMA authorisedAcid beta-glucosidase deficiency; CYP2D6 metabolizer status for eliglustat; Long-term treatment of Gaucher disease; Cerdelga switch/maintenance context after disease control on ERT in pediatric EU wording; UK HST material for type 1 Gaucher disease treatment pathway. · EMA and NICE sources do not equal automatic access in each EU member state. NICE material is reuse-restricted and should be linked rather than quoted extensively. France/Germany reimbursement should be verified in later cells. Confidence/conflicts: High for EMA/NICE pathway statements; medium for current UK access details because the fetched NICE document is a consultation document rather than the final guidance page.
  • Imiglucerase (Cerezyme); eliglustat (Cerdelga); miglustat (Zavesca); enzyme replacement therapy including imiglucerase or velaglucerase alfa[2]EMA authorisedAcid beta-glucosidase deficiency; CYP2D6 metabolizer status for eliglustat; Long-term treatment of Gaucher disease; Cerdelga switch/maintenance context after disease control on ERT in pediatric EU wording; UK HST material for type 1 Gaucher disease treatment pathway. · EMA and NICE sources do not equal automatic access in each EU member state. NICE material is reuse-restricted and should be linked rather than quoted extensively. France/Germany reimbursement should be verified in later cells. Confidence/conflicts: High for EMA/NICE pathway statements; medium for current UK access details because the fetched NICE document is a consultation document rather than the final guidance page.
  • Imiglucerase (Cerezyme); eliglustat (Cerdelga); miglustat (Zavesca); enzyme replacement therapy including imiglucerase or velaglucerase alfa[2]EMA authorisedAcid beta-glucosidase deficiency; CYP2D6 metabolizer status for eliglustat; Long-term treatment of Gaucher disease; Cerdelga switch/maintenance context after disease control on ERT in pediatric EU wording; UK HST material for type 1 Gaucher disease treatment pathway. · EMA and NICE sources do not equal automatic access in each EU member state. NICE material is reuse-restricted and should be linked rather than quoted extensively. France/Germany reimbursement should be verified in later cells. Confidence/conflicts: High for EMA/NICE pathway statements; medium for current UK access details because the fetched NICE document is a consultation document rather than the final guidance page.

Japan

  • eliglustat (Cerdelga 100 mg capsule)[3]PMDA-approved (Japan)CYP2D6 metabolizer phenotype and hepatic impairment affect eliglustat eligibility/contraindications per source; GBA genotype not specified in fetched indication text; oral substrate reduction therapy; PMDA review discusses Gaucher disease type 1 evidence, including patients with prior enzyme replacement therapy and treatment-naive adults. · The PMDA safety summary lists CYP2D6 metabolizer/hepatic impairment contraindication revisions and dosage-adjustment language. PMDA's review notes eliglustat was clinically developed for Gaucher disease type 1 and had not been used in patients with types 2 and 3 or pediatric patients in the development program; neurologic symptoms are not established as a target. This entry does not establish reimbursement or full current package insert details. Confidence/conflicts: High for Japanese Cerdelga indication and PMDA review context; no conflict identified. Full current package insert and reimbursement remain gaps.
  • imiglucerase, genetically recombinant (Cerezyme for i.v. injection 400 units)[4]PMDA-approved (Japan)acid beta-glucosidase / glucocerebrosidase deficiency context; source does not specify genotype; enzyme replacement therapy for symptomatic Gaucher disease manifestations; source does not limit the indication by Gaucher type in the fetched text. · The PMDA document is a safety-revision summary adding infusion reaction to clinically significant adverse reactions and important precautions. It confirms indication and market launch but does not provide full dosing, reimbursement, neurologic-effectiveness limitations, or center access details. Japanese full package insert should be checked before patient-facing reuse. Confidence/conflicts: High for Japanese Cerezyme indication and market-launch details from PMDA; no conflict identified. Full label, reimbursement, and neurologic limitations remain gaps.

Australia

  • eliglustat (Cerdelga)[5]TGA-registered (Australia)CYP2D6 metabolizer status affects eliglustat use and dose; adult Gaucher disease type 1 substrate-reduction therapy; Australian LSDP access for eligible type 1 Gaucher disease. · TGA AusPAR is from the 2015 approval review and points users to current PI for the latest label. LSDP guideline limits subsidised treatment to type 1 Gaucher disease and includes program eligibility criteria; it does not establish type 2 or type 3 access. Confidence/conflicts: High for Australian approval and LSDP listing; current product-information details still should be checked before patient-facing reuse. Availability/reimbursement outside the approving regulator not established.
  • imiglucerase (Cerezyme); velaglucerase (VPRIV); taliglucerase (Elelyso); eliglustat (Cerdelga)[6]Approvedglucocerebrosidase deficiency / GBA-related Gaucher disease context; CYP2D6 metabolizer status relevant for eliglustat; Australian LSDP-subsidised treatment for eligible Gaucher disease type 1 patients; treating physicians request the most appropriate medicine where eligibility requirements are met. · Subsidised treatment through the LSDP is not available for type 2 or type 3 Gaucher disease. Eligibility, ongoing reapplication, clinical stability, treatment switching, and monitoring rules are program-specific. Confidence/conflicts: High for Australian LSDP type 1 access and listed medicines; no conflict identified. Availability/reimbursement outside the approving regulator not established.
  • imiglucerase (Cerezyme); velaglucerase (VPRIV); taliglucerase (Elelyso); eliglustat (Cerdelga)[6]Approvedglucocerebrosidase deficiency / GBA-related Gaucher disease context; CYP2D6 metabolizer status relevant for eliglustat; Australian LSDP-subsidised treatment for eligible Gaucher disease type 1 patients; treating physicians request the most appropriate medicine where eligibility requirements are met. · Subsidised treatment through the LSDP is not available for type 2 or type 3 Gaucher disease. Eligibility, ongoing reapplication, clinical stability, treatment switching, and monitoring rules are program-specific. Confidence/conflicts: High for Australian LSDP type 1 access and listed medicines; no conflict identified. Availability/reimbursement outside the approving regulator not established.

Thailand

  • imiglucerase (Cerezyme)[7]EMA authorisedglucocerebrosidase enzyme deficiency or pathogenic GBA1 mutation; absence of severe neurologic features of Gaucher type 2/3 per access criteria; controlled-use/preauthorization access for Gaucher disease type 1; source names Cerezyme in the imiglucerase dosing section. · Thai-language clinical/access criteria require human review before patient-facing reuse. The source excludes patients with severe neurologic findings characteristic of Gaucher type 2/3 from this access pathway and ties continued therapy to follow-up/assessment rules. This is an access criteria source, not a full Thai product label. Confidence/conflicts: Medium-high for existence of a Thai controlled-use pathway; no direct conflict identified, but Thai-language clinical details need human review before reuse. Primary source is in Thai. English summary pending human review — confirm exact wording with your care team. Availability/reimbursement outside the approving regulator not established.
  • velaglucerase alfa (VPRIV)[8]Approvedconfirmed Gaucher disease; neurologic manifestations relevant; long-term enzyme replacement therapy for confirmed Gaucher disease; source states patients currently treated with imiglucerase ERT for type 1 Gaucher disease may be switched to VPRIV using the same dose/frequency. · The source says VPRIV should be administered only to patients with confirmed Gaucher disease and that effectiveness on neurological symptoms is not expected. This finding does not establish reimbursement, public-program eligibility, or stock availability. Confidence/conflicts: High for Thai VPRIV label/authorization details; no conflict identified, but payer status remains unresolved.

Canada

  • eliglustat (Cerdelga)[9]Health Canada approvedCYP2D6 poor, intermediate, or extensive metabolizer status by genotype testing; ultra-rapid and indeterminate metabolizer limitations; adult type 1 Gaucher disease oral substrate-reduction therapy after CYP2D6 genotyping; Canadian reimbursement-with-conditions context. · Safety and effectiveness in pediatric patients under 18 have not been established in the SBD. CADTH reimbursement recommendation is not the same as automatic provincial or territorial coverage. Confidence/conflicts: High for Canadian label and CADTH reimbursement-condition context; no conflict identified, but payer implementation is jurisdiction-specific.
  • imiglucerase (Cerezyme)[10]Approvedconfirmed non-neuronopathic type 1 or chronic neuronopathic type 3 Gaucher disease with non-neurological manifestations; long-term enzyme replacement therapy for non-neurological manifestations of type 1 or type 3 Gaucher disease in Canada. · The monograph notes hypersensitivity/anaphylaxis risks, infusion monitoring, and that efficacy for neurological symptoms in chronic neuronopathic Gaucher disease has not been established. It is not a provincial reimbursement rule. Confidence/conflicts: High for Canadian label indication and neurologic caveat; no conflict identified.
  • imiglucerase (Cerezyme)[10]Approvedconfirmed non-neuronopathic type 1 or chronic neuronopathic type 3 Gaucher disease with non-neurological manifestations; long-term enzyme replacement therapy for non-neurological manifestations of type 1 or type 3 Gaucher disease in Canada. · The monograph notes hypersensitivity/anaphylaxis risks, infusion monitoring, and that efficacy for neurological symptoms in chronic neuronopathic Gaucher disease has not been established. It is not a provincial reimbursement rule. Confidence/conflicts: High for Canadian label indication and neurologic caveat; no conflict identified.
  • velaglucerase alfa (VPRIV)[11]Approvedglucocerebrosidase deficiency / type 1 Gaucher disease context; long-term ERT for pediatric and adult type 1 Gaucher disease in Canada. · The monograph verifies Health Canada label context but not provincial reimbursement or comparative preference among ERTs. Infusion/hypersensitivity monitoring and product-specific precautions remain clinical-team responsibilities. Confidence/conflicts: High for Canadian VPRIV label indication; no conflict identified.
  • velaglucerase alfa (VPRIV)[11]Approvedglucocerebrosidase deficiency / type 1 Gaucher disease context; long-term ERT for pediatric and adult type 1 Gaucher disease in Canada. · The monograph verifies Health Canada label context but not provincial reimbursement or comparative preference among ERTs. Infusion/hypersensitivity monitoring and product-specific precautions remain clinical-team responsibilities. Confidence/conflicts: High for Canadian VPRIV label indication; no conflict identified.

Sources

  1. U.S. Food and Drug Administration — official drug label · official drug label
  2. European Medicines Agency (EMA) — regulator EPAR · regulator EPAR
  3. Pharmaceuticals and Medical Devices Agency (PMDA) — safety investigation / label revision summary PDF · safety investigation / label revision summary PDF
  4. Pharmaceuticals and Medical Devices Agency (PMDA) — safety investigation / label revision summary PDF · safety investigation / label revision summary PDF
  5. Therapeutic Goods Administration (TGA) — Australian Public Assessment Report · Australian Public Assessment Report
  6. Australian Government Department of Health, Disability and Ageing — LSDP resource collection · LSDP resource collection
  7. Thai National Drug Information / Thai FDA-MOPH — national essential medicines controlled-use/access criteria PDF · national essential medicines controlled-use/access criteria PDF
  8. Thai National Drug Information / Thai FDA-MOPH — SmPC PDF / regulator drug-information repository · SmPC PDF / regulator drug-information repository
  9. Health Canada product monograph repository / Sanofi-aventis Canada — official product monograph · official product monograph
  10. Sanofi-aventis Canada / Health Canada product monograph — official product monograph · official product monograph
  11. Health Canada product monograph repository / Takeda Canada — official product monograph · official product monograph

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

  • Eliglustat; imiglucerase (Cerezyme); velaglucerase alfa (VPRIV); venglustatClinical trialClinical trialReported in a clinical trialJapan · Acid beta-glucosidase deficiency; GBA genotype; CYP2D6 metabolism for eliglustat; Japanese ERT access context; pediatric Gaucher type 1/type 3 eliglustat safety/PK; adult and pediatric type 3 venglustat studies with background imiglucerase/ERT. · Venglustat is investigational in the fetched records. PMDA discussion is a review report and should be supplemented by current Japanese package inserts in a later pass. Russia/France/Germany/UK trial participation does not prove reimbursement or routine availability. Confidence/conflicts: High for Japan PMDA historical approval context and registry geography; routine access in Russia/China/France/Germany/UK remains source-pending outside the trial records. Pharmaceuticals and Medical Devices Agency (PMDA) — regulator review report
  • Eliglustat; imiglucerase (Cerezyme); velaglucerase alfa (VPRIV); venglustatClinical trialClinical trialReported in a clinical trialJapan · Acid beta-glucosidase deficiency; GBA genotype; CYP2D6 metabolism for eliglustat; Japanese ERT access context; pediatric Gaucher type 1/type 3 eliglustat safety/PK; adult and pediatric type 3 venglustat studies with background imiglucerase/ERT. · Venglustat is investigational in the fetched records. PMDA discussion is a review report and should be supplemented by current Japanese package inserts in a later pass. Russia/France/Germany/UK trial participation does not prove reimbursement or routine availability. Confidence/conflicts: High for Japan PMDA historical approval context and registry geography; routine access in Russia/China/France/Germany/UK remains source-pending outside the trial records. Pharmaceuticals and Medical Devices Agency (PMDA) — regulator review report
  • Eliglustat; imiglucerase (Cerezyme); velaglucerase alfa (VPRIV); venglustatClinical trialClinical trialReported in a clinical trialJapan · Acid beta-glucosidase deficiency; GBA genotype; CYP2D6 metabolism for eliglustat; Japanese ERT access context; pediatric Gaucher type 1/type 3 eliglustat safety/PK; adult and pediatric type 3 venglustat studies with background imiglucerase/ERT. · Venglustat is investigational in the fetched records. PMDA discussion is a review report and should be supplemented by current Japanese package inserts in a later pass. Russia/France/Germany/UK trial participation does not prove reimbursement or routine availability. Confidence/conflicts: High for Japan PMDA historical approval context and registry geography; routine access in Russia/China/France/Germany/UK remains source-pending outside the trial records. Pharmaceuticals and Medical Devices Agency (PMDA) — regulator review report
  • Velaglucerase alfa (VPRIV); imiglucerase comparator; eliglustat; ICGG Gaucher Registry observationClinical trial · NCT00635427Clinical trialTrial only (registry)Korea · Confirmed beta-glucocerebrosidase deficiency and/or GBA mutation per registry eligibility; Type 1 Gaucher ERT extension and comparative studies; observational registry tracking routine outcomes irrespective of treatment status. · Registry inclusion indicates documented diagnosis and treating-physician-determined care, not a specific drug approval. Thailand direct treatment options remain source-pending. Older completed trials should not be treated as current access. Confidence/conflicts: High for registry/trial geography; medium-low for treatment availability in Thailand and Russia because official regulator/reimbursement sources remain unverified. ClinicalTrials.gov — clinical-trial registry
  • Velaglucerase alfa (VPRIV); imiglucerase comparator; eliglustat; ICGG Gaucher Registry observationClinical trial · NCT00635427Clinical trialTrial only (registry)Korea · Confirmed beta-glucocerebrosidase deficiency and/or GBA mutation per registry eligibility; Type 1 Gaucher ERT extension and comparative studies; observational registry tracking routine outcomes irrespective of treatment status. · Registry inclusion indicates documented diagnosis and treating-physician-determined care, not a specific drug approval. Thailand direct treatment options remain source-pending. Older completed trials should not be treated as current access. Confidence/conflicts: High for registry/trial geography; medium-low for treatment availability in Thailand and Russia because official regulator/reimbursement sources remain unverified. ClinicalTrials.gov — clinical-trial registry
  • Velaglucerase alfa (VPRIV); imiglucerase comparator; eliglustat; ICGG Gaucher Registry observationClinical trial · NCT00635427Clinical trialTrial only (registry)Korea · Confirmed beta-glucocerebrosidase deficiency and/or GBA mutation per registry eligibility; Type 1 Gaucher ERT extension and comparative studies; observational registry tracking routine outcomes irrespective of treatment status. · Registry inclusion indicates documented diagnosis and treating-physician-determined care, not a specific drug approval. Thailand direct treatment options remain source-pending. Older completed trials should not be treated as current access. Confidence/conflicts: High for registry/trial geography; medium-low for treatment availability in Thailand and Russia because official regulator/reimbursement sources remain unverified. ClinicalTrials.gov — clinical-trial registry
  • Velaglucerase alfa (VPRIV); imiglucerase comparator; eliglustat; ICGG Gaucher Registry observationClinical trial · NCT00635427Clinical trialTrial only (registry)Korea · Confirmed beta-glucocerebrosidase deficiency and/or GBA mutation per registry eligibility; Type 1 Gaucher ERT extension and comparative studies; observational registry tracking routine outcomes irrespective of treatment status. · Registry inclusion indicates documented diagnosis and treating-physician-determined care, not a specific drug approval. Thailand direct treatment options remain source-pending. Older completed trials should not be treated as current access. Confidence/conflicts: High for registry/trial geography; medium-low for treatment availability in Thailand and Russia because official regulator/reimbursement sources remain unverified. ClinicalTrials.gov — clinical-trial registry
  • LY-M001; VGN-R08b; imiglucerase (Cerezyme); CAN103; venglustatClinical trial · NCT06818838Clinical trialTrial only (registry)China · GBA1 biallelic mutations; low glucocerebrosidase/GCase activity; Adult type 1 Gaucher gene-therapy trial; pediatric/adolescent type 1 gene-therapy trial; infant type 2 investigational treatment; type 3 imiglucerase study; type 3 venglustat study. · Registry status varies across recruiting, active-not-recruiting, completed, and unknown. NMPA labels and reimbursement were not verified in this cell. CAN103 and VGN-R08b remain investigational from the fetched sources. Confidence/conflicts: High for clinical-trial geography and status; low for routine availability because no NMPA label was verified this cycle. ClinicalTrials.gov — clinical-trial registry
  • LY-M001; VGN-R08b; imiglucerase (Cerezyme); CAN103; venglustatClinical trial · NCT06818838Clinical trialTrial only (registry)China · GBA1 biallelic mutations; low glucocerebrosidase/GCase activity; Adult type 1 Gaucher gene-therapy trial; pediatric/adolescent type 1 gene-therapy trial; infant type 2 investigational treatment; type 3 imiglucerase study; type 3 venglustat study. · Registry status varies across recruiting, active-not-recruiting, completed, and unknown. NMPA labels and reimbursement were not verified in this cell. CAN103 and VGN-R08b remain investigational from the fetched sources. Confidence/conflicts: High for clinical-trial geography and status; low for routine availability because no NMPA label was verified this cycle. ClinicalTrials.gov — clinical-trial registry
  • LY-M001; VGN-R08b; imiglucerase (Cerezyme); CAN103; venglustatClinical trial · NCT06818838Clinical trialTrial only (registry)China · GBA1 biallelic mutations; low glucocerebrosidase/GCase activity; Adult type 1 Gaucher gene-therapy trial; pediatric/adolescent type 1 gene-therapy trial; infant type 2 investigational treatment; type 3 imiglucerase study; type 3 venglustat study. · Registry status varies across recruiting, active-not-recruiting, completed, and unknown. NMPA labels and reimbursement were not verified in this cell. CAN103 and VGN-R08b remain investigational from the fetched sources. Confidence/conflicts: High for clinical-trial geography and status; low for routine availability because no NMPA label was verified this cycle. ClinicalTrials.gov — clinical-trial registry

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.