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Gaucher disease: 국가별 선택지

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선택지 정리됨희귀·유전 질환최종 확인 2026.06

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임상시험 검색어

Gaucher disease clinical trial

국가별 선택지

국가별 치료 선택지

공식 규제·평가 기관 출처를 바탕으로 한 국가별 승인·접근 상태입니다. 무엇이 어디에 존재하는지를 보여줄 뿐, 추천이 아닙니다.

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.

출처

  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

위 내용은 공식 규제·접근 상태일 뿐, 의학적 조언이나 추천이 아니고, 적격성을 판단하지도 않습니다. 어떤 선택지가 적합한지는 환자의 상황과 종양내과 팀에 달려 있습니다. 규제 상태는 바뀔 수 있으니 표시된 출처에서 확인하세요. 임상 세부 내용은 영문이 정본입니다. 최종 확인 2026-06-12.