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선택지 정리됨

Paroxysmal nocturnal hemoglobinuria (PNH): 국가별 선택지

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선택지 정리됨희귀·유전 질환최종 확인 2026.06

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임상시험 검색어

Paroxysmal nocturnal hemoglobinuria (PNH) 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

  • eculizumab (Soliris); ravulizumab (Ultomiris); pegcetacoplan (Empaveli); iptacopan (Fabhalta); crovalimab-akkz (Piasky); danicopan (Voydeya)[1]FDA-approvedPNH clone / complement-mediated hemolysis; extravascular hemolysis subset when specified; Complement inhibition for PNH hemolysis; proximal complement inhibition in adults; Voydeya specifically as add-on for extravascular hemolysis while on ravulizumab or eculizumab. · Complement inhibitors carry serious infection/vaccination/REMS warnings in the labels. PNH indication scopes vary by age, weight, monotherapy versus add-on status, and intravascular versus extravascular hemolysis. No sequencing or superiority claim is made. Confidence/conflicts: High for U.S. label claims; no conflict identified.
  • eculizumab (Soliris); ravulizumab (Ultomiris); pegcetacoplan (Empaveli); iptacopan (Fabhalta); crovalimab-akkz (Piasky); danicopan (Voydeya)[1]FDA-approvedPNH clone / complement-mediated hemolysis; extravascular hemolysis subset when specified; Complement inhibition for PNH hemolysis; proximal complement inhibition in adults; Voydeya specifically as add-on for extravascular hemolysis while on ravulizumab or eculizumab. · Complement inhibitors carry serious infection/vaccination/REMS warnings in the labels. PNH indication scopes vary by age, weight, monotherapy versus add-on status, and intravascular versus extravascular hemolysis. No sequencing or superiority claim is made. Confidence/conflicts: High for U.S. label claims; no conflict identified.

European Union

  • crovalimab (Piasky); danicopan (Voydeya)[2]EMA authorisedcomplement-mediated hemolysis; residual hemolytic anemia/extravascular hemolysis when specified; Piasky C5 inhibitor treatment in PNH; Voydeya add-on for residual hemolytic anemia/EVH while on eculizumab or ravulizumab; China crovalimab for complement-inhibitor-naive adults/adolescents 12+ per Chugai. · China and Japan Piasky/Voydeya claims are manufacturer/regulatory-announcement sourced except EMA Piasky. EU Voydeya source is AstraZeneca announcement, not EMA EPAR in this pass. National reimbursement is not established. Confidence/conflicts: High for EMA Piasky; medium for Japan/China/EU Voydeya and China/Japan Piasky until direct PMDA/NMPA/EMA product pages are fetched.
  • crovalimab (Piasky); danicopan (Voydeya)[2]EMA authorisedcomplement-mediated hemolysis; residual hemolytic anemia/extravascular hemolysis when specified; Piasky C5 inhibitor treatment in PNH; Voydeya add-on for residual hemolytic anemia/EVH while on eculizumab or ravulizumab; China crovalimab for complement-inhibitor-naive adults/adolescents 12+ per Chugai. · China and Japan Piasky/Voydeya claims are manufacturer/regulatory-announcement sourced except EMA Piasky. EU Voydeya source is AstraZeneca announcement, not EMA EPAR in this pass. National reimbursement is not established. Confidence/conflicts: High for EMA Piasky; medium for Japan/China/EU Voydeya and China/Japan Piasky until direct PMDA/NMPA/EMA product pages are fetched.

Australia

  • eculizumab (Soliris); ravulizumab (Ultomiris); pegcetacoplan (Empaveli)[3]TGA-registered (Australia)PNH clone / complement-mediated hemolysis; PIGA somatic mutation context described by source; C5 inhibition for PNH hemolysis; ravulizumab for adult/paediatric PNH body weight 10 kg or above in stated settings; pegcetacoplan after inadequate response to or intolerance of a C5 inhibitor. · The AusPAR summarizes Australian regulator status and indication context but is not a payer document. Complement inhibitor infection/vaccination precautions require current product information and local hematology review. Confidence/conflicts: High for Australian PNH approval context for Soliris, Ultomiris, and Empaveli; direct current PI and PBS criteria remain source-pending. No conflict identified. Availability/reimbursement outside the approving regulator not established.
  • iptacopan (Fabhalta)[4]TGA-registered (Australia)PNH clone / complement-mediated hemolysis; adult PNH in Australia. · The TGA AusPAR verifies registration and approved indication but not PBS subsidy or sequencing versus C5 inhibitors or pegcetacoplan. Infection prevention requirements should be checked in the current PI. Confidence/conflicts: High for Australian registration and indication; payer status remains source-pending. No conflict identified. Availability/reimbursement outside the approving regulator not established.

Thailand

  • ravulizumab (Ultomiris)[5]Approvedcomplement-mediated hemolysis context; source defines PNH treatment population by hemolysis with clinical symptoms indicating high disease activity, or clinical stability after eculizumab for at least 6 months; complement-inhibitor-naive PNH with high disease activity symptoms, or switch/maintenance context after at least 6 months of eculizumab stability; adult and pediatric patients weighing at least 10 kg. · Ravulizumab must be administered by a healthcare professional under supervision of a physician experienced in hematological disorders. The label states PNH is chronic and ravulizumab treatment is recommended to continue for the patient's lifetime unless discontinuation is clinically indicated. It also states pediatric PNH patients under 30 kg were not studied and that dosing for those patients is extrapolated from aHUS PK/PD data. Meningococcal vaccination/prophylaxis is described in the clinical-study context; this entry does not establish Thai reimbursement, center availability, or whether another complement inhibitor is preferred. Confidence/conflicts: High for Thai NDI Ultomiris PNH label content; no conflict identified. Thai reimbursement/access and direct Thai marketing-authorization metadata remain unresolved.

Canada

  • crovalimab (Piasky)[6]Health Canada approvedPNH clone / complement-mediated hemolysis; PNH in adults and adolescents 13 years and older weighing at least 40 kg in Canada. · The indication does not establish payer coverage, self-administration suitability, or switching criteria. Complement inhibition infection risk and vaccination requirements should be confirmed from the current monograph and care team. Confidence/conflicts: High for Canadian crovalimab indication; reimbursement and access pathway remain source-pending. No conflict identified.
  • danicopan (Voydeya) add-on to ravulizumab or eculizumab[7]ApprovedPNH clone; EVH while on terminal complement inhibition; adult PNH with residual hemolytic anemia due to EVH as add-on to ravulizumab or eculizumab. · The monograph states Health Canada has not authorized pediatric use. CDA-AMC conditions include prior C5 inhibitor reimbursement criteria, stable C5 inhibitor treatment, persistent anemia due to EVH, reticulocyte threshold, hematologist prescribing, and price/implementation conditions; provincial adoption remains source-pending. Confidence/conflicts: High for Canadian add-on indication and CDA-AMC conditional recommendation; provincial implementation remains source-pending. No conflict identified.
  • iptacopan (Fabhalta)[8]Health Canada approvedPNH clone / complement-mediated hemolysis; adult PNH with hemolytic anemia in Canada. · Health Canada indication is monotherapy and adult-only. The product monograph includes contraindications and serious infection precautions; reimbursement and provincial availability were not established by these sources. Confidence/conflicts: High for Canadian approval and label; reimbursement remains source-pending. 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. Therapeutic Goods Administration (TGA) — Australian Public Assessment Report · Australian Public Assessment Report
  4. Therapeutic Goods Administration (TGA) — Australian Public Assessment Report · Australian Public Assessment Report
  5. Thai National Drug Information / Thai FDA-MOPH — SmPC PDF / regulator drug-information repository · SmPC PDF / regulator drug-information repository
  6. Health Canada product monograph repository / Roche Canada — official product monograph · official product monograph
  7. Alexion Canada / Health Canada product monograph — official product monograph · official product monograph
  8. 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.