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Fabry disease: 국가별 선택지

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선택지 정리됨희귀·유전 질환최종 확인 2026.06

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임상시험 검색어

Fabry 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

  • Agalsidase beta (Fabrazyme); migalastat (Galafold); pegunigalsidase alfa-iwxj (Elfabrio)[1]FDA accelerated approvalGLA variant / alpha-galactosidase A deficiency; amenable GLA variant for migalastat; Enzyme replacement therapy for confirmed Fabry disease; oral pharmacologic chaperone for adults with amenable GLA variants; adult ERT for Elfabrio. · Fabrazyme and Elfabrio require infusion-supervision and monitoring for hypersensitivity/infusion reactions. Galafold is limited to adults with amenable pathogenic/likely pathogenic GLA variants and FDA notes accelerated approval may depend on confirmatory clinical benefit. Confidence/conflicts: High for FDA-labeled U.S. options. No claim is made about superiority, eligibility, or payer access.
  • Agalsidase beta (Fabrazyme); migalastat (Galafold); pegunigalsidase alfa-iwxj (Elfabrio)[1]FDA accelerated approvalGLA variant / alpha-galactosidase A deficiency; amenable GLA variant for migalastat; Enzyme replacement therapy for confirmed Fabry disease; oral pharmacologic chaperone for adults with amenable GLA variants; adult ERT for Elfabrio. · Fabrazyme and Elfabrio require infusion-supervision and monitoring for hypersensitivity/infusion reactions. Galafold is limited to adults with amenable pathogenic/likely pathogenic GLA variants and FDA notes accelerated approval may depend on confirmatory clinical benefit. Confidence/conflicts: High for FDA-labeled U.S. options. No claim is made about superiority, eligibility, or payer access.

European Union

  • Agalsidase beta (Fabrazyme); agalsidase alfa (Replagal); migalastat (Galafold); pegunigalsidase alfa (Elfabrio)[2]EMA authorisedAlpha-galactosidase A deficiency; amenable mutation for Galafold; Long-term ERT and disease-modifying therapy; adult pegunigalsidase alfa under NICE conditions; Galafold only for amenable mutations. · EMA authorization does not equal automatic access in every EU member state. NICE is England-specific and reuse-restricted/link-only. France/Germany reimbursement is not yet verified. Confidence/conflicts: High for EMA and NICE statements; country-level France/Germany reimbursement remains unresolved.
  • Agalsidase beta (Fabrazyme); agalsidase alfa (Replagal); migalastat (Galafold); pegunigalsidase alfa (Elfabrio)[2]EMA authorisedAlpha-galactosidase A deficiency; amenable mutation for Galafold; Long-term ERT and disease-modifying therapy; adult pegunigalsidase alfa under NICE conditions; Galafold only for amenable mutations. · EMA authorization does not equal automatic access in every EU member state. NICE is England-specific and reuse-restricted/link-only. France/Germany reimbursement is not yet verified. Confidence/conflicts: High for EMA and NICE statements; country-level France/Germany reimbursement remains unresolved.

Japan

  • agalsidase beta, genetically recombinant (Fabrazyme; Agalsidase Beta BS I.V. Infusion [JCR])[3]PMDA-approved (Japan)alpha-galactosidase A deficiency / GLA-related Fabry context; source does not specify genotype; enzyme replacement therapy for Fabry disease; source is a biosimilar approval list and does not further specify phenotype, organ involvement, or line of therapy. · This source verifies Japanese approval/indication for the agalsidase beta biosimilar and reference product context, but it is not the full Fabrazyme or biosimilar package insert. It does not establish reimbursement, infusion-center access, antibody/infusion-reaction management, renal/cardiac endpoints, or pediatric/adult details. Confidence/conflicts: High for Japanese agalsidase beta biosimilar approval and Fabry indication; no conflict identified. Full label and reimbursement remain gaps.
  • migalastat (Galafold 123 mg capsule)[4]PMDA-approved (Japan)GLA mutation amenable to migalastat / Galafold; confirmed Fabry diagnosis; oral pharmacological chaperone/precision-medicine option for Fabry disease patients aged 16 years and older with amenable GLA mutations. · The MHLW approval statement is manufacturer-reported; PMDA source text includes Japanese/foreign-label application material and should be checked against the current Japanese package insert before patient-facing reuse. Galafold is mutation-specific and not a general Fabry therapy for non-amenable mutations. This entry does not establish current reimbursement, launch status, or comparative choice versus enzyme replacement therapy. Confidence/conflicts: Medium-high for Japanese Galafold approval and amenable-mutation indication; no conflict identified. Current Japanese package insert and reimbursement remain gaps.

Australia

  • agalsidase alfa (Replagal); agalsidase beta (Fabrazyme)[5]TGA-registered (Australia)alpha-galactosidase A deficiency or GLA mutation confirming Fabry disease; Australian LSDP-subsidised enzyme replacement therapy for eligible confirmed Fabry disease. · LSDP eligibility requires periodic evaluation, specialist physician application, Medicare citizenship/residency criteria, and ongoing eligibility. Home infusion requires prior hospital infusions and stability assessment. The source notes Galafold is now PBS rather than LSDP. Confidence/conflicts: High for Australian LSDP ERT access; no conflict identified. confirm current TGA ARTG registration status Availability/reimbursement outside the approving regulator not established.
  • agalsidase alfa (Replagal); agalsidase beta (Fabrazyme)[5]TGA-registered (Australia)alpha-galactosidase A deficiency or GLA mutation confirming Fabry disease; Australian LSDP-subsidised enzyme replacement therapy for eligible confirmed Fabry disease. · LSDP eligibility requires periodic evaluation, specialist physician application, Medicare citizenship/residency criteria, and ongoing eligibility. Home infusion requires prior hospital infusions and stability assessment. The source notes Galafold is now PBS rather than LSDP. Confidence/conflicts: High for Australian LSDP ERT access; no conflict identified. confirm current TGA ARTG registration status Availability/reimbursement outside the approving regulator not established.
  • migalastat (Galafold)[6]TGA-registered (Australia)amenable GLA mutation / alpha-galactosidase A deficiency; oral pharmacologic chaperone therapy for confirmed Fabry disease with amenable mutation; Australian PBS access context noted by LSDP guideline. · The TGA AusPAR age wording is 16 years and older at the time of the 2017 review, while the LSDP guideline only states PBS availability and does not reproduce full PBS criteria. Current PI/PBS restrictions should be checked for age, amenable-variant list, and access criteria. Confidence/conflicts: Medium-high; approval and PBS-transfer context verified, but current PBS criteria remain source-pending. confirm current TGA ARTG registration status Availability/reimbursement outside the approving regulator not established.
  • pegunigalsidase alfa (Elfabrio)[7]TGA-registered (Australia)confirmed Fabry disease / alpha-galactosidase A deficiency; adult confirmed Fabry disease enzyme replacement therapy in Australia. · The AusPAR discusses a small rare-disease safety dataset and notes no pediatric safety data were presented for this application. The source verifies TGA registration but not PBS or LSDP funding. Confidence/conflicts: High for TGA registration and indication; funding/access status source-pending. confirm current TGA ARTG registration status Availability/reimbursement outside the approving regulator not established.
  • pegunigalsidase alfa (Elfabrio)[7]TGA-registered (Australia)confirmed Fabry disease / alpha-galactosidase A deficiency; adult confirmed Fabry disease enzyme replacement therapy in Australia. · The AusPAR discusses a small rare-disease safety dataset and notes no pediatric safety data were presented for this application. The source verifies TGA registration but not PBS or LSDP funding. Confidence/conflicts: High for TGA registration and indication; funding/access status source-pending. confirm current TGA ARTG registration status Availability/reimbursement outside the approving regulator not established.

Thailand

  • agalsidase alfa (Replagal)[8]Approvedconfirmed alpha-galactosidase A deficiency / Fabry disease; long-term enzyme replacement therapy for confirmed Fabry disease. · The label states treatment should be supervised by a physician experienced in Fabry disease or other inherited metabolic diseases. It notes extensive renal damage may limit renal response to ERT, includes infusion-related and hypersensitivity/anaphylaxis warnings, and does not establish reimbursement or stock availability in Thailand. Confidence/conflicts: High for Thai Replagal label and authorization details; no conflict identified, but payer/access status remains a gap.

Canada

  • agalsidase beta (Fabrazyme)[9]Approvedconfirmed Fabry disease; long-term enzyme replacement therapy for confirmed Fabry disease in Canada. · The monograph verifies Health Canada label context but not provincial reimbursement. It includes infusion and hypersensitivity considerations that require clinical oversight. Confidence/conflicts: High for Canadian Fabrazyme label indication; no conflict identified.
  • agalsidase beta (Fabrazyme)[9]Approvedconfirmed Fabry disease; long-term enzyme replacement therapy for confirmed Fabry disease in Canada. · The monograph verifies Health Canada label context but not provincial reimbursement. It includes infusion and hypersensitivity considerations that require clinical oversight. Confidence/conflicts: High for Canadian Fabrazyme label indication; no conflict identified.
  • migalastat (Galafold); pegunigalsidase alfa (Elfabrio)[10]Health Canada approvedamenable alpha-galactosidase A mutation for migalastat; confirmed alpha-galactosidase A deficiency for Elfabrio; Galafold oral chaperone therapy for amenable Fabry mutations age 12+; Elfabrio adult ERT and Canadian reimbursement-with-conditions context. · Galafold should not be used for non-amenable mutations, severe renal insufficiency per label, or concomitantly with ERT. Elfabrio RDS highlights uncertainty in treatment-naive patients and primary safety concerns of severe hypersensitivity/anaphylaxis and infusion-associated reactions. CDA-AMC recommendation does not equal automatic provincial implementation. Confidence/conflicts: High for Galafold and Elfabrio Canadian regulatory status; medium-high for reimbursement because provincial implementation remains a separate step.
  • migalastat (Galafold); pegunigalsidase alfa (Elfabrio)[10]Health Canada approvedamenable alpha-galactosidase A mutation for migalastat; confirmed alpha-galactosidase A deficiency for Elfabrio; Galafold oral chaperone therapy for amenable Fabry mutations age 12+; Elfabrio adult ERT and Canadian reimbursement-with-conditions context. · Galafold should not be used for non-amenable mutations, severe renal insufficiency per label, or concomitantly with ERT. Elfabrio RDS highlights uncertainty in treatment-naive patients and primary safety concerns of severe hypersensitivity/anaphylaxis and infusion-associated reactions. CDA-AMC recommendation does not equal automatic provincial implementation. Confidence/conflicts: High for Galafold and Elfabrio Canadian regulatory status; medium-high for reimbursement because provincial implementation remains a separate step.

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) — approved biosimilars list PDF · approved biosimilars list PDF
  4. Amicus Therapeutics via SEC exhibit — manufacturer regulatory approval announcement · manufacturer regulatory approval announcement
  5. Australian Government Department of Health and Aged Care — LSDP clinical access guideline · LSDP clinical access guideline
  6. Therapeutic Goods Administration (TGA) — Australian Public Assessment Report · Australian Public Assessment Report
  7. Therapeutic Goods Administration (TGA) — Australian Public Assessment Report · Australian Public Assessment Report
  8. Thai National Drug Information / Thai FDA-MOPH — SmPC PDF / regulator drug-information repository · SmPC PDF / regulator drug-information repository
  9. Sanofi-aventis Canada / Health Canada product monograph — official product monograph · official product monograph
  10. Amicus Therapeutics Canada / Health Canada product monograph — 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.