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낭성 섬유증: 국가별 치료 선택지
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선택지 정리됨희귀·유전 질환최종 확인 2026.06
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
- Ivacaftor / Kalydeco; lumacaftor-ivacaftor / Orkambi; tezacaftor-ivacaftor / Symdeko; elexacaftor-tezacaftor-ivacaftor / Trikafta; vanzacaftor-tezacaftor-deutivacaftor / Alyftrek[1]FDA-approvedCFTR mutations responsive to each modulator; at least one F508del or responsive mutation for Trikafta/Alyftrek per FDA expansion; genotype confirmation needed when unknown; Mutation-directed CFTR modulator therapy for people with CF meeting age and genotype criteria for each product. · CFTR modulators are genotype- and age-specific. Access, prior authorization, liver monitoring, drug interactions, and whether two modulators may be combined require clinician/pharmacy review. Confidence/conflicts: High for U.S. modulator approval landscape; Vertex expansion announcement is manufacturer source and should be paired with label review when integrating.
European Union
- Kalydeco, Orkambi, Symkevi/Symdeko, Kaftrio/Trikafta, Alyftrek[2]EMA authorisedCFTR mutation responsiveness; F508del and other non-class I/responsive mutation classes vary by EMA product; Mutation-directed CFTR modulator therapy in eligible people with CF, with national payer criteria for NHS England. · EMA authorization does not set reimbursement in every member state. NICE/NHS England sources apply to England and have commercial-arrangement and commissioning details. Confidence/conflicts: High for EU/UK access claims; no conflict identified.
Australia
- elexacaftor/tezacaftor/ivacaftor and ivacaftor (Trikafta)[3]TGA-registered (Australia)at least one F508del mutation in CFTR; CFTR-modulator treatment for CF patients aged 12 years and older with at least one F508del mutation. · This source records the original Australian regulatory decision and not current PBS criteria, later pediatric/rare-mutation label extensions, liver monitoring requirements, or individual suitability. Confidence/conflicts: Medium-high for original Australian Trikafta approval; later age/mutation expansions require separate current-label/PBS follow-up. Availability/reimbursement outside the approving regulator not established.
- ivacaftor (Kalydeco)[4]TGA-registered (Australia)CFTR mutations responsive to ivacaftor potentiation based on clinical and/or in vitro assay data; CFTR potentiator therapy for CF patients aged 4 months and older with an ivacaftor-responsive CFTR mutation. · The AusPAR states the product information approved with that submission may have been superseded and directs readers to the TGA PI/CMI search facility for the most recent PI. This entry does not establish PBS access or a specific mutation's eligibility. Confidence/conflicts: High for the Australian 2024 Kalydeco extension described in the AusPAR; current PI/PBS implementation remains a follow-up. Availability/reimbursement outside the approving regulator not established.
- vanzacaftor/tezacaftor/deutivacaftor (Alyftrek)[5]TGA-registered (Australia)broad CFTR mutation-responsive context, including F508del and other responsive mutations per source; CFTR-modulator treatment for people aged 6 years and older with cystic fibrosis and relevant CFTR mutation context. · The TGA public summary describes broad mutation coverage but does not enumerate all eligible mutations in the fetched page. It does not establish PBS subsidy or whether an individual mutation is included. Confidence/conflicts: Medium-high for Australian Alyftrek registration; mutation list and payer criteria remain source-pending. Availability/reimbursement outside the approving regulator not established.
Canada
- elexacaftor/tezacaftor/ivacaftor and ivacaftor (Trikafta)[6]Health Canada approvedat least one F508del mutation in CFTR; later Health Canada materials also describe responsive-mutation expansion; Canadian CFTR-modulator treatment for CF patients aged 2 years and older within indicated mutation groups. · The product monograph states genotype confirmation should be performed if genotype is unknown and notes no pediatric authorization below age 2. This entry separates regulatory authorization from provincial or private payer access. Confidence/conflicts: Medium-high; current monograph and later RDS support broader/pediatric context, while the original SBD reflects the older 12+ indication. Availability/reimbursement outside the approving regulator not established.
- elexacaftor/tezacaftor/ivacaftor and ivacaftor (Trikafta)[7]CADTH reimbursement recommendationresponsive CFTR mutation context; reimbursement review specifies 152 non-F508del mutations and broader responsive-mutation framing; Canadian public-drug-plan reimbursement-with-conditions context for Trikafta in responsive CFTR mutations. · CDA-AMC recommendations are not the same as Health Canada authorization or provincial implementation. Conditions include evidence of benefit after a defined period and a price reduction; actual access depends on the relevant public or private plan. Confidence/conflicts: Medium-high for Canadian reimbursement recommendation; jurisdictional plan adoption remains source-pending.
- vanzacaftor/tezacaftor/deutivacaftor (Alyftrek)[8]Health Canada approvedat least one F508del mutation or another responsive CFTR mutation; Canadian CFTR-modulator treatment and reimbursement-with-conditions context for patients aged 6 years and older with F508del or responsive CFTR mutations. · Health Canada authorization does not guarantee public-plan coverage. CDA-AMC notes cost and adoption uncertainties, and its recommendation includes specialist prescribing and cost conditions. Confidence/conflicts: High for Canadian authorization plus reimbursement recommendation; provincial implementation and mutation-list details remain follow-up.
출처
- U.S. Food and Drug Administration — official drug label · official drug label
- European Medicines Agency (EMA) — regulator EPAR · regulator EPAR
- Therapeutic Goods Administration (TGA) — Australian Prescription Medicine Decision Summary · Australian Prescription Medicine Decision Summary
- Therapeutic Goods Administration (TGA) — Australian Public Assessment Report · Australian Public Assessment Report
- Therapeutic Goods Administration (TGA) — Australian Prescription Medicine Decision Summary · Australian Prescription Medicine Decision Summary
- Health Canada / Vertex Canada product monograph repository — official product monograph · official product monograph
- CDA-AMC / CADTH via NCBI Bookshelf — reimbursement recommendation / health technology review · reimbursement recommendation / health technology review
- Health Canada Drug and Health Product 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.
승인된 치료 너머
임상시험 및 신흥 선택지
환자가 거주하는 국가에서 아직 승인된 표준 치료가 아닌 선택지입니다 — 연구, 임상시험, 허가 외 사용, 담당 종양내과 의사가 먼저 언급하지 않을 수 있는 초기 근거입니다. 각 항목은 근거의 강도에 따라 구분됩니다. 여기에 실린 항목은 조사하고 의료진과 상의할 정보일 뿐, 효과가 입증되었거나 안전하거나 현재 이용 가능하다는 의미가 아닙니다. 임상 세부 내용은 영문이 정본입니다.
임상시험 진행 중
- VX-522 mRNA therapy with ivacaftor; dirocaftor/posenacaftor/nesolicaftor (diponecaftor in registry); CTX-4430; PTI-428임상시험 · NCT05668741임상시험Trial only (registry)United States · CFTR genotypes not responsive to CFTR modulator therapy for VX-522; HIT-CF/organoid study completion for dirocaftor/posenacaftor/nesolicaftor trial; confirmed CF for CTX-4430/PTI-428 studies; Adults with CF not responsive to CFTR modulators for VX-522; adult CF study populations for other investigational agents. · Several records are completed; VX-522 is active-not-recruiting. Investigational mRNA/modulator approaches should not be presented as approved access. Confidence/conflicts: High for registry cells; no approval claim is made. ClinicalTrials.gov — clinical-trial registry
- Elexacaftor-tezacaftor-ivacaftor / Kaftrio-Trikafta; initiation of CFTR modulator therapy임상시험 · NCT05699148임상시험Trial only (registry)United Kingdom · At least one Phe508del mutation in UK pediatric Kaftrio gut-imaging record; partial-function or N1303K CFTR in U.S. Trikafta study; Children aged 6-11 years with CF and at least one Phe508del mutation in the UK registry record; genotype expansion/functional testing contexts in U.S. records. · The UK study registry statement is not a regulator label. U.S. study of mutations not approved at that time should be checked against current expanded FDA labels. Confidence/conflicts: Medium-high; registry statements are verified but should be paired with current regulator label for patient-facing integration. ClinicalTrials.gov — clinical-trial registry
임상시험이나 초기 보고에 실렸다는 것은 해당 선택지가 연구되고 있다는 의미일 뿐, 효과가 있거나 특정 환자에게 안전하거나 현재 등록 가능하다는 뜻은 아닙니다. 어떤 선택지가 적합한지는 담당 종양내과 팀과 임상시험 팀이 함께 상의할 문제입니다. 최종 확인 2026.06.
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모든 선택지는 규제·평가·지침 등 공신력 있는 출처에 연결되며, 새 출처가 검증되면 계속 확장됩니다.
진료 전 정리할 정보
- Which CFTR mutations are documented, and are they listed as responsive for the proposed modulator?
- Is the proposed option older modulator therapy, Trikafta, or Alyftrek?
- What liver, eye, drug-interaction, and pregnancy considerations apply locally?
- Is access based on EMA label, NICE TA988, NHS England commissioning, or another national payer?