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선택지 정리됨

Hemophilia A / B: 국가별 선택지

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선택지 정리됨희귀·유전 질환최종 확인 2026.06

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임상시험 검색어

Hemophilia A / B 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

  • Valoctocogene roxaparvovec / Roctavian; etranacogene dezaparvovec / Hemgenix; fidanacogene elaparvovec / Beqvez; marstacimab / Hympavzi; concizumab / Alhemo[1]FDA-approvedHemophilia A severe FVIII deficiency for Roctavian; hemophilia B factor IX deficiency for Hemgenix/Beqvez; AAV antibody and inhibitor exclusions vary by product; Adults with severe hemophilia A for Roctavian; adults with hemophilia B for Hemgenix/Beqvez contexts; hemophilia A/B prophylaxis or treatment settings per anti-TFPI product label/news. · Gene therapy requires specialist eligibility evaluation, AAV antibody testing, liver monitoring, and long-term follow-up. Beqvez U.S. approval and commercialization discontinuation both need to be visible when discussing access. Confidence/conflicts: Medium-high. Roctavian is FDA primary; Hemgenix/Beqvez/Hympavzi need direct FDA labels in a later pass; Beqvez access is affected by discontinuation reporting. AVAILABILITY NOTE: Beqvez (fidanacogene elaparvovec) was FDA-approved for hemophilia B in April 2024, but Pfizer discontinued its commercialization globally in February 2025 and halted production; no patients received it commercially and the EU MA was withdrawn 15 May 2025. The FDA approval was not revoked, but Beqvez cannot currently be purchased or obtained in the US. The available hemophilia B gene therapy is Hemgenix (etranacogene dezaparvovec). Do not pursue Beqvez as a live option. Roctavian/Hemgenix/Hympavzi/Alhemo remain approved and available.
  • Valoctocogene roxaparvovec / Roctavian; etranacogene dezaparvovec / Hemgenix; fidanacogene elaparvovec / Beqvez; marstacimab / Hympavzi; concizumab / Alhemo[1]FDA-approvedHemophilia A severe FVIII deficiency for Roctavian; hemophilia B factor IX deficiency for Hemgenix/Beqvez; AAV antibody and inhibitor exclusions vary by product; Adults with severe hemophilia A for Roctavian; adults with hemophilia B for Hemgenix/Beqvez contexts; hemophilia A/B prophylaxis or treatment settings per anti-TFPI product label/news. · Gene therapy requires specialist eligibility evaluation, AAV antibody testing, liver monitoring, and long-term follow-up. Beqvez U.S. approval and commercialization discontinuation both need to be visible when discussing access. Confidence/conflicts: Medium-high. Roctavian is FDA primary; Hemgenix/Beqvez/Hympavzi need direct FDA labels in a later pass; Beqvez access is affected by discontinuation reporting. AVAILABILITY NOTE: Beqvez (fidanacogene elaparvovec) was FDA-approved for hemophilia B in April 2024, but Pfizer discontinued its commercialization globally in February 2025 and halted production; no patients received it commercially and the EU MA was withdrawn 15 May 2025. The FDA approval was not revoked, but Beqvez cannot currently be purchased or obtained in the US. The available hemophilia B gene therapy is Hemgenix (etranacogene dezaparvovec). Do not pursue Beqvez as a live option. Roctavian/Hemgenix/Hympavzi/Alhemo remain approved and available.
  • Valoctocogene roxaparvovec / Roctavian; etranacogene dezaparvovec / Hemgenix; fidanacogene elaparvovec / Beqvez; marstacimab / Hympavzi; concizumab / Alhemo[1]FDA-approvedHemophilia A severe FVIII deficiency for Roctavian; hemophilia B factor IX deficiency for Hemgenix/Beqvez; AAV antibody and inhibitor exclusions vary by product; Adults with severe hemophilia A for Roctavian; adults with hemophilia B for Hemgenix/Beqvez contexts; hemophilia A/B prophylaxis or treatment settings per anti-TFPI product label/news. · Gene therapy requires specialist eligibility evaluation, AAV antibody testing, liver monitoring, and long-term follow-up. Beqvez U.S. approval and commercialization discontinuation both need to be visible when discussing access. Confidence/conflicts: Medium-high. Roctavian is FDA primary; Hemgenix/Beqvez/Hympavzi need direct FDA labels in a later pass; Beqvez access is affected by discontinuation reporting. AVAILABILITY NOTE: Beqvez (fidanacogene elaparvovec) was FDA-approved for hemophilia B in April 2024, but Pfizer discontinued its commercialization globally in February 2025 and halted production; no patients received it commercially and the EU MA was withdrawn 15 May 2025. The FDA approval was not revoked, but Beqvez cannot currently be purchased or obtained in the US. The available hemophilia B gene therapy is Hemgenix (etranacogene dezaparvovec). Do not pursue Beqvez as a live option. Roctavian/Hemgenix/Hympavzi/Alhemo remain approved and available.

European Union

  • Hemgenix; Durveqtix; Roctavian; Hympavzi; etranacogene dezaparvovec[2]Approved (restricted indication)Factor VIII or IX deficiency; inhibitor status and AAV antibody exclusions vary by therapy; Adult hemophilia B gene therapy for Hemgenix/Hemgenix NICE managed access; severe/moderately severe hemophilia B for Durveqtix; hemophilia A/B anti-TFPI context for Hympavzi. · EMA authorization and NICE managed access have distinct access implications. Manufacturer statements for Hympavzi should be verified against EMA EPAR in a later pass. Confidence/conflicts: High for Hemgenix/NICE; medium for Hympavzi EU detail pending primary EPAR capture.
  • Hemgenix; Durveqtix; Roctavian; Hympavzi; etranacogene dezaparvovec[2]Approved (restricted indication)Factor VIII or IX deficiency; inhibitor status and AAV antibody exclusions vary by therapy; Adult hemophilia B gene therapy for Hemgenix/Hemgenix NICE managed access; severe/moderately severe hemophilia B for Durveqtix; hemophilia A/B anti-TFPI context for Hympavzi. · EMA authorization and NICE managed access have distinct access implications. Manufacturer statements for Hympavzi should be verified against EMA EPAR in a later pass. Confidence/conflicts: High for Hemgenix/NICE; medium for Hympavzi EU detail pending primary EPAR capture.

Japan

  • concizumab (Alhemo)[3]PMDA-approved (Japan)factor VIII inhibitor or factor IX inhibitor; control of bleeding tendency in congenital hemophilia with factor VIII or IX inhibitors; source specifies patients aged 12 years or older in the dosage section. · The PMDA English translation states the Japanese original takes precedence. PMDA notes very limited numbers of Japanese patients treated before approval and requires postmarketing survey. The review also discusses thromboembolic events and need for risk-mitigation information around breakthrough bleeding. Confidence/conflicts: High for Japan PMDA/MHLW approval context; no conflict identified, with translation caveat that Japanese original prevails. Availability/reimbursement outside the approving regulator not established.
  • emicizumab (Hemlibra)[4]PMDA-approved (Japan)congenital blood coagulation factor VIII deficiency with blood coagulation factor VIII inhibitors; reduction in bleeding tendency in congenital factor VIII deficiency with factor VIII inhibitors, as stated in the PMDA review report. · The PMDA English translation states the Japanese original takes precedence. The approval conditions require a risk management plan and postmarketing use-results survey because only limited Japanese patients had received the product before launch. Confidence/conflicts: High for Japan PMDA/MHLW approval context; no conflict identified, with translation caveat that Japanese original prevails. Availability/reimbursement outside the approving regulator not established.
  • emicizumab (Hemlibra)[5]Approvedacquired factor VIII inhibitors/autoantibodies described by source; routine prophylaxis to prevent or reduce bleeding episodes in acquired hemophilia A, as stated in the Japanese approval notice. · This source is a manufacturer regulatory news release rather than the PMDA review report. It is not a reimbursement determination and is not a patient-specific eligibility statement. Confidence/conflicts: Medium-high; direct MHLW approval claim from the marketing authorization holder was verified, but PMDA label/review text for this extension was not captured in this finding.
  • marstacimab (Hympavzi)[6]PMDA-approved (Japan)absence of factor VIII or IX inhibitors; age and weight criteria appear in the source dosage section; routine prophylaxis/control of bleeding tendency in congenital hemophilia without factor VIII or IX inhibitors; the dosage section specifies patients aged 12 years or older weighing at least 35 kg. · The PMDA English translation states the Japanese original takes precedence. The review includes approval conditions for risk-management planning and all-patient postmarketing survey. Confidence/conflicts: High for Japan PMDA review-report indication; no conflict identified, with translation caveat that Japanese original prevails. Availability/reimbursement outside the approving regulator not established.
  • nonacog beta pegol (Refixia)[7]PMDA-approved (Japan)factor IX deficiency; source notes hemophilia B FIX activity criteria in clinical-study context; control of bleeding tendency in factor IX deficiency, including treatment of bleeding episodes, treatment in surgery, and routine prophylaxis, as stated in the PMDA review report. · The PMDA English translation states the Japanese original takes precedence. PMDA states safety and efficacy in clinical practice should be further evaluated in postmarketing clinical studies and surveillance, including long-term exposure considerations. Confidence/conflicts: High for Japan PMDA/MHLW approval context; no conflict identified, with translation caveat that Japanese original prevails. Availability/reimbursement outside the approving regulator not established.

Australia

  • emicizumab (Hemlibra)[8]TGA-registered (Australia)congenital factor VIII deficiency; factor VIII inhibitor status relevant; routine prophylaxis for adult and paediatric haemophilia A; Australian national blood arrangement access through specialist haemophilia centres. · The NBA page describes national supply and specialist-centre access rather than individual eligibility. TGA indication and NBA funding/access criteria should be checked against current local haemophilia centre policy before reuse. Confidence/conflicts: High for Australian TGA indication and NBA national-supply context; no conflict identified. Availability/reimbursement outside the approving regulator not established.
  • emicizumab (Hemlibra)[8]TGA-registered (Australia)congenital factor VIII deficiency; factor VIII inhibitor status relevant; routine prophylaxis for adult and paediatric haemophilia A; Australian national blood arrangement access through specialist haemophilia centres. · The NBA page describes national supply and specialist-centre access rather than individual eligibility. TGA indication and NBA funding/access criteria should be checked against current local haemophilia centre policy before reuse. Confidence/conflicts: High for Australian TGA indication and NBA national-supply context; no conflict identified. Availability/reimbursement outside the approving regulator not established.
  • etranacogene dezaparvovec (Hemgenix)[9]TGA-registered (Australia)congenital factor IX deficiency; factor IX inhibitor history and AAV-related gene-therapy considerations relevant; adult haemophilia B gene therapy for people meeting the TGA-stated prophylaxis or bleeding-history criteria. · TGA states the provisional approval was based on short-term efficacy and safety data, with continued approval dependent on confirmation of longer-term benefit from ongoing clinical trials. The source does not establish reimbursement, treatment-centre capacity, or individual eligibility. Confidence/conflicts: High for Australian provisional approval and indication; reimbursement/access status remains a gap.
  • etranacogene dezaparvovec (Hemgenix)[9]TGA-registered (Australia)congenital factor IX deficiency; factor IX inhibitor history and AAV-related gene-therapy considerations relevant; adult haemophilia B gene therapy for people meeting the TGA-stated prophylaxis or bleeding-history criteria. · TGA states the provisional approval was based on short-term efficacy and safety data, with continued approval dependent on confirmation of longer-term benefit from ongoing clinical trials. The source does not establish reimbursement, treatment-centre capacity, or individual eligibility. Confidence/conflicts: High for Australian provisional approval and indication; reimbursement/access status remains a gap.

Thailand

  • activated prothrombin complex concentrate / factor eight inhibitor bypassing activity (FEIBA)[10]Approvedfactor VIII inhibitor for hemophilia A; factor IX inhibitor for hemophilia B; inhibitor-associated bleeding treatment/prophylaxis and surgical prophylaxis contexts as stated in the Thai NDI label. · The label notes limited experience in children under 6 years and limited clinical data for prophylaxis in hemophilia B patients with inhibitors; it also includes thromboembolic warnings. This entry does not establish Thai reimbursement or availability. Confidence/conflicts: High for Thai label indications; no conflict identified in fetched source, with explicit caveat that hemophilia B prophylaxis data are limited.
  • emicizumab (Hemlibra)[11]Approvedcongenital factor VIII deficiency; factor VIII inhibitor status relevant; routine prophylaxis for hemophilia A with or without factor VIII inhibitors, as stated in the Thai NDI label. · The label states treatment should be initiated under supervision of a physician experienced in hemophilia and/or bleeding disorders, and that bypassing agents should be discontinued the day before starting Hemlibra therapy; this queue entry does not establish reimbursement or center availability in Thailand. Confidence/conflicts: High for Thai label indication and setting; no conflict identified in fetched source, but Thai reimbursement status was not verified in this finding.
  • moroctocog alfa / antihemophilic factor recombinant (Xyntha)[12]EMA authorisedcongenital factor VIII deficiency; von Willebrand disease exclusion relevant; on-demand bleeding treatment/control, perioperative management, and routine prophylaxis for hemophilia A, as stated in the Thai NDI label. · The label states Xyntha does not contain von Willebrand factor and is not indicated for von Willebrand disease; dosing and duration depend on factor VIII deficiency severity, bleed location/extent, and clinical condition. This entry does not establish reimbursement or stock availability. Confidence/conflicts: High for Thai registration/label indication; no source conflict identified.
  • nonacog alfa / recombinant coagulation factor IX (BeneFIX)[13]EMA authorisedcongenital factor IX deficiency; factor IX inhibitor status relevant; on-demand bleeding treatment/control, perioperative management, and routine prophylaxis for hemophilia B, as stated in the Thai NDI label. · The label states BeneFIX is not indicated for induction of immune tolerance in patients with hemophilia B; it also states treatment should be supervised by a physician experienced in hemophilia B and monitored with factor IX activity assays where possible. This entry does not establish Thai reimbursement or stock availability. Confidence/conflicts: High for Thai label indication; no source conflict identified.

Canada

  • concizumab (Alhemo)[14]Health Canada approvedcongenital factor IX deficiency with FIX inhibitors; routine prophylaxis for adolescent and adult hemophilia B patients with factor IX inhibitors in Canada. · The Canadian product monograph source captured here is for hemophilia B with FIX inhibitors; it does not establish a Canadian indication for hemophilia A or for hemophilia B without inhibitors. The monograph includes thromboembolic-event warnings and instructions to discontinue prophylactic bypassing agents before initiation. Confidence/conflicts: High for Canadian hemophilia B with FIX inhibitor label; no source conflict identified, but broader hemophilia A/B claims from non-Canadian sources were not recorded. Availability/reimbursement outside the approving regulator not established.
  • concizumab (Alhemo)[14]Health Canada approvedcongenital factor IX deficiency with FIX inhibitors; routine prophylaxis for adolescent and adult hemophilia B patients with factor IX inhibitors in Canada. · The Canadian product monograph source captured here is for hemophilia B with FIX inhibitors; it does not establish a Canadian indication for hemophilia A or for hemophilia B without inhibitors. The monograph includes thromboembolic-event warnings and instructions to discontinue prophylactic bypassing agents before initiation. Confidence/conflicts: High for Canadian hemophilia B with FIX inhibitor label; no source conflict identified, but broader hemophilia A/B claims from non-Canadian sources were not recorded. Availability/reimbursement outside the approving regulator not established.
  • emicizumab (Hemlibra)[15]Health Canada approvedcongenital factor VIII deficiency; factor VIII inhibitor status relevant; routine prophylaxis for hemophilia A with or without factor VIII inhibitors in Canada. · The product monograph is a label, not a provincial reimbursement decision or individual access pathway. It includes safety warnings and administration requirements that must be reviewed by the treating team. Confidence/conflicts: High for Canadian label indication; no reimbursement conclusion recorded from this source.
  • emicizumab (Hemlibra)[15]Health Canada approvedcongenital factor VIII deficiency; factor VIII inhibitor status relevant; routine prophylaxis for hemophilia A with or without factor VIII inhibitors in Canada. · The product monograph is a label, not a provincial reimbursement decision or individual access pathway. It includes safety warnings and administration requirements that must be reviewed by the treating team. Confidence/conflicts: High for Canadian label indication; no reimbursement conclusion recorded from this source.
  • etranacogene dezaparvovec (Hemgenix)[16]Health Canada approvedcongenital factor IX deficiency; AAV5 neutralizing antibody threshold, factor IX inhibitor status, liver status, and sex/pediatric status relevant per label; adult hemophilia B requiring routine prophylaxis; reimbursement conditions focus on moderately severe to severe hemophilia B with defined FIX activity and AAV5 criteria. · Health Canada notes no clinical experience in mild or moderate hemophilia B with FIX activity over 2%. CADTH/CDA-AMC reimbursement recommendations are not the same as automatic provincial coverage or treatment-centre availability. Confidence/conflicts: High for Health Canada approval and label constraints; medium-high for reimbursement context because implementation remains jurisdiction-specific.
  • etranacogene dezaparvovec (Hemgenix)[16]Health Canada approvedcongenital factor IX deficiency; AAV5 neutralizing antibody threshold, factor IX inhibitor status, liver status, and sex/pediatric status relevant per label; adult hemophilia B requiring routine prophylaxis; reimbursement conditions focus on moderately severe to severe hemophilia B with defined FIX activity and AAV5 criteria. · Health Canada notes no clinical experience in mild or moderate hemophilia B with FIX activity over 2%. CADTH/CDA-AMC reimbursement recommendations are not the same as automatic provincial coverage or treatment-centre availability. Confidence/conflicts: High for Health Canada approval and label constraints; medium-high for reimbursement context because implementation remains jurisdiction-specific.

출처

  1. U.S. Food and Drug Administration — regulator approval notice · regulator approval notice
  2. European Medicines Agency (EMA) — regulator EPAR · regulator EPAR
  3. Pharmaceuticals and Medical Devices Agency / Ministry of Health, Labour and Welfare — regulator review report PDF · regulator review report PDF
  4. Pharmaceuticals and Medical Devices Agency / Ministry of Health, Labour and Welfare — regulator review report PDF · regulator review report PDF
  5. Chugai Pharmaceutical Co., Ltd. — manufacturer regulatory approval notice · manufacturer regulatory approval notice
  6. Pharmaceuticals and Medical Devices Agency / Ministry of Health, Labour and Welfare — regulator review report PDF · regulator review report PDF
  7. Pharmaceuticals and Medical Devices Agency / Ministry of Health, Labour and Welfare — regulator review report PDF · regulator review report PDF
  8. Therapeutic Goods Administration (TGA) — prescription medicine registration · prescription medicine registration
  9. Therapeutic Goods Administration (TGA) — Australian prescription medicine decision summary · Australian prescription medicine decision summary
  10. Thai National Drug Information / Thai FDA-MOPH — SmPC PDF / regulator drug-information repository · SmPC PDF / regulator drug-information repository
  11. Thai National Drug Information / Thai FDA-MOPH — SmPC PDF / regulator drug-information repository · SmPC PDF / regulator drug-information repository
  12. Thai National Drug Information / Thai FDA-MOPH — SmPC PDF / regulator drug-information repository · SmPC PDF / regulator drug-information repository
  13. Thai National Drug Information / Thai FDA-MOPH — SmPC PDF / regulator drug-information repository · SmPC PDF / regulator drug-information repository
  14. Novo Nordisk Canada / Health Canada product monograph — official product monograph · official product monograph
  15. Hoffmann-La Roche Limited / Health Canada product monograph — official product monograph · official product monograph
  16. Health Canada Drug and Health Products Portal — Summary Basis of Decision · Summary Basis of Decision

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.