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Spinal muscular atrophy (SMA): 국가별 선택지

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선택지 정리됨희귀·유전 질환최종 확인 2026.06

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임상시험 검색어

Spinal muscular atrophy (SMA) 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

  • Nusinersen / Spinraza; risdiplam / Evrysdi; onasemnogene abeparvovec-xioi / Zolgensma; onasemnogene abeparvovec-brve / Itvisma[1]FDA-approvedSMN1 mutation or bi-allelic SMN1 mutation for gene therapy; SMN2 copy and age restrictions vary by product/source; Pediatric and adult SMA for nusinersen; patients 2 months and older for risdiplam; pediatric patients younger than 2 years for IV Zolgensma; adult and pediatric patients aged 2 years and older for intrathecal Itvisma. · Gene-therapy products have age, mutation, administration-route, and safety-monitoring requirements. FDA sources do not determine payer coverage or individual eligibility. Confidence/conflicts: High for U.S. approval status and label-scope summaries. No source conflict identified.
  • Nusinersen / Spinraza; risdiplam / Evrysdi; onasemnogene abeparvovec-xioi / Zolgensma; onasemnogene abeparvovec-brve / Itvisma[1]FDA-approvedSMN1 mutation or bi-allelic SMN1 mutation for gene therapy; SMN2 copy and age restrictions vary by product/source; Pediatric and adult SMA for nusinersen; patients 2 months and older for risdiplam; pediatric patients younger than 2 years for IV Zolgensma; adult and pediatric patients aged 2 years and older for intrathecal Itvisma. · Gene-therapy products have age, mutation, administration-route, and safety-monitoring requirements. FDA sources do not determine payer coverage or individual eligibility. Confidence/conflicts: High for U.S. approval status and label-scope summaries. No source conflict identified.

European Union

  • Nusinersen / Spinraza; risdiplam / Evrysdi; onasemnogene abeparvovec / Zolgensma[2]Approved (restricted indication)5q SMA; SMN1 inherited mutations for Zolgensma; SMN2 copy/clinical type restrictions per EMA product pages; EMA-labeled disease-modifying therapy for 5q SMA; NHS routine-access and presymptomatic gene-therapy appraisal contexts as specified by NICE. · EMA authorization does not establish price/reimbursement in every EU member state. NICE final draft guidance and HST guidance apply to England/NHS appraisal scope and are not identical to EU or devolved-country access. Confidence/conflicts: High for EMA and NICE statements; no conflict identified, but NICE scope is national and conditional by recommendation wording.
  • Nusinersen / Spinraza; risdiplam / Evrysdi; onasemnogene abeparvovec / Zolgensma[2]Approved (restricted indication)5q SMA; SMN1 inherited mutations for Zolgensma; SMN2 copy/clinical type restrictions per EMA product pages; EMA-labeled disease-modifying therapy for 5q SMA; NHS routine-access and presymptomatic gene-therapy appraisal contexts as specified by NICE. · EMA authorization does not establish price/reimbursement in every EU member state. NICE final draft guidance and HST guidance apply to England/NHS appraisal scope and are not identical to EU or devolved-country access. Confidence/conflicts: High for EMA and NICE statements; no conflict identified, but NICE scope is national and conditional by recommendation wording.

Japan

  • Onasemnogene abeparvovec / Zolgensma; risdiplam / Evrysdi; nusinersen noted in PMDA review context[3]PMDA-approved (Japan)Bi-allelic SMN1 deletion or mutation for Zolgensma; anti-AAV9 antibody negative condition; SMA indication for risdiplam; Zolgensma in symptomatic or presymptomatic SMA patients younger than 2 years with bi-allelic SMN1 mutation/deletion and anti-AAV9 antibody criteria; Evrysdi for SMA across infants, children, and adults as described by Chugai announcement. · Novartis and Chugai announcements are manufacturer sources; PMDA report is authoritative for Zolgensma. Primary PMDA risdiplam label remains an active gap. Confidence/conflicts: High for Japan Zolgensma via PMDA; medium-high for Evrysdi because source is manufacturer announcement naming MHLW approval. Availability/reimbursement outside the approving regulator not established.
  • Onasemnogene abeparvovec / Zolgensma; risdiplam / Evrysdi; nusinersen noted in PMDA review context[3]PMDA-approved (Japan)Bi-allelic SMN1 deletion or mutation for Zolgensma; anti-AAV9 antibody negative condition; SMA indication for risdiplam; Zolgensma in symptomatic or presymptomatic SMA patients younger than 2 years with bi-allelic SMN1 mutation/deletion and anti-AAV9 antibody criteria; Evrysdi for SMA across infants, children, and adults as described by Chugai announcement. · Novartis and Chugai announcements are manufacturer sources; PMDA report is authoritative for Zolgensma. Primary PMDA risdiplam label remains an active gap. Confidence/conflicts: High for Japan Zolgensma via PMDA; medium-high for Evrysdi because source is manufacturer announcement naming MHLW approval. Availability/reimbursement outside the approving regulator not established.

Australia

  • nusinersen (Spinraza)[4]TGA-registered (Australia)5q SMA / SMN1-related disease context; SMN2-targeted treatment mechanism; 5q SMA treatment in Australia; intrathecal therapy context should be checked against current Australian product information and payer criteria. · AusPARs are static documents tied to a submission at a particular point in time. This entry records regulator assessment context, not current PBS criteria, intrathecal procedural suitability, or individual eligibility. Confidence/conflicts: Medium-high for Australian nusinersen regulatory context; current PI/PBS implementation remains a follow-up. Availability/reimbursement outside the approving regulator not established.
  • nusinersen (Spinraza)[4]TGA-registered (Australia)5q SMA / SMN1-related disease context; SMN2-targeted treatment mechanism; 5q SMA treatment in Australia; intrathecal therapy context should be checked against current Australian product information and payer criteria. · AusPARs are static documents tied to a submission at a particular point in time. This entry records regulator assessment context, not current PBS criteria, intrathecal procedural suitability, or individual eligibility. Confidence/conflicts: Medium-high for Australian nusinersen regulatory context; current PI/PBS implementation remains a follow-up. Availability/reimbursement outside the approving regulator not established.
  • nusinersen (Spinraza)[4]TGA-registered (Australia)5q SMA / SMN1-related disease context; SMN2-targeted treatment mechanism; 5q SMA treatment in Australia; intrathecal therapy context should be checked against current Australian product information and payer criteria. · AusPARs are static documents tied to a submission at a particular point in time. This entry records regulator assessment context, not current PBS criteria, intrathecal procedural suitability, or individual eligibility. Confidence/conflicts: Medium-high for Australian nusinersen regulatory context; current PI/PBS implementation remains a follow-up. Availability/reimbursement outside the approving regulator not established.
  • risdiplam (Evrysdi)[5]TGA-registered (Australia)5q SMA / SMN2 splicing modifier context; 5q SMA in patients aged 2 months and older in Australia. · The TGA page records regulatory status and mechanism context, not PBS subsidy, newborn-screening pathway, or individual suitability. Confidence/conflicts: High for Australian Evrysdi regulatory status; no conflict identified. Availability/reimbursement outside the approving regulator not established.
  • risdiplam (Evrysdi)[5]TGA-registered (Australia)5q SMA / SMN2 splicing modifier context; 5q SMA in patients aged 2 months and older in Australia. · The TGA page records regulatory status and mechanism context, not PBS subsidy, newborn-screening pathway, or individual suitability. Confidence/conflicts: High for Australian Evrysdi regulatory status; no conflict identified. Availability/reimbursement outside the approving regulator not established.

Canada

  • nusinersen (Spinraza)[6]Health Canada approved5q SMA / SMN2-targeted antisense oligonucleotide context; Canadian 5q SMA treatment context, including pediatric evidence; monograph notes limited data over age 18. · The monograph states overall management still requires strategies for motor decline, respiratory complications, and feeding difficulties. It specifies intrathecal lumbar-puncture administration by experienced professionals. Confidence/conflicts: High for Canadian Spinraza label/regulatory context; no conflict identified. Availability/reimbursement outside the approving regulator not established.
  • nusinersen (Spinraza)[6]Health Canada approved5q SMA / SMN2-targeted antisense oligonucleotide context; Canadian 5q SMA treatment context, including pediatric evidence; monograph notes limited data over age 18. · The monograph states overall management still requires strategies for motor decline, respiratory complications, and feeding difficulties. It specifies intrathecal lumbar-puncture administration by experienced professionals. Confidence/conflicts: High for Canadian Spinraza label/regulatory context; no conflict identified. Availability/reimbursement outside the approving regulator not established.
  • nusinersen (Spinraza)[6]Health Canada approved5q SMA / SMN2-targeted antisense oligonucleotide context; Canadian 5q SMA treatment context, including pediatric evidence; monograph notes limited data over age 18. · The monograph states overall management still requires strategies for motor decline, respiratory complications, and feeding difficulties. It specifies intrathecal lumbar-puncture administration by experienced professionals. Confidence/conflicts: High for Canadian Spinraza label/regulatory context; no conflict identified. Availability/reimbursement outside the approving regulator not established.
  • risdiplam (Evrysdi)[7]Health Canada approvedSMN1/SMN2 disease mechanism context; Canadian SMA treatment context; Health Canada SBD specifies patients 2 months and older. · The product monograph includes oral/enteral formulation details and notes limited data in older adults; this entry does not establish payer coverage or choice among SMA disease-modifying therapies. Confidence/conflicts: High for Canadian Evrysdi label/regulatory context; no conflict identified. Availability/reimbursement outside the approving regulator not established.
  • risdiplam (Evrysdi)[7]Health Canada approvedSMN1/SMN2 disease mechanism context; Canadian SMA treatment context; Health Canada SBD specifies patients 2 months and older. · The product monograph includes oral/enteral formulation details and notes limited data in older adults; this entry does not establish payer coverage or choice among SMA disease-modifying therapies. Confidence/conflicts: High for Canadian Evrysdi label/regulatory context; no conflict identified. Availability/reimbursement outside the approving regulator not established.

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 / MHLW — regulator review report · regulator review report
  4. Therapeutic Goods Administration (TGA) — Australian Public Assessment Report · Australian Public Assessment Report
  5. Therapeutic Goods Administration (TGA) — Australian Prescription Medicine Decision Summary · Australian Prescription Medicine Decision Summary
  6. Biogen Canada / Health Canada product monograph format — official product monograph · official product monograph
  7. Hoffmann-La Roche Canada / Health Canada product monograph format — 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.