Options mapped

Cystic fibrosis: options by country

Sourced options by country plus visit-prep questions for Cystic fibrosis. Each line links to its regulator, HTA, or guideline source. This page maps options; it does not recommend or rank them.

Options mappedRare diseaseLast checked June 2026

What this page does

Maps options by country

It maps sourced options by country alongside diagnosis wording, stage, test results, specialists, and trial-search terms.

What it does not do

Does not choose treatment

It does not rank treatments, recommend a choice, or decide clinical fit.

Where it comes from

Built on trusted sources

Every option links to a trusted regulator, HTA, or guideline source, and the list grows as new sources pass verification.

Information to gather before the next visit

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?

Trial-search terms to discuss

Cystic fibrosis clinical trial

Options by country

Treatments by country

Regulatory and access status by country, from official sources. It shows what exists and where — not a recommendation.

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.
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.
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.
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)[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.
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.
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.

Sources

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

This is official regulatory and access status only — not medical advice, not a recommendation, and not a statement about eligibility. Whether any option fits depends on your situation and your oncology team. Status changes over time; confirm the current position with the linked source. Last checked 2026-06-12.

Beyond approved care

In clinical trials & emerging options

Options that are not — or not yet — an approved standard where you live: studies, clinical trials, off-label use, and early evidence that your own oncologist may not raise. Each is labeled by how strong the evidence is. A listing here is information to research and discuss with your team; it does not mean a treatment is proven, safe for you, or available today.

In clinical trials

VX-522 mRNA therapy with ivacaftor; dirocaftor/posenacaftor/nesolicaftor (diponecaftor in registry); CTX-4430; PTI-428Clinical trial · NCT05668741Clinical trialTrial 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
VX-522 mRNA therapy with ivacaftor; dirocaftor/posenacaftor/nesolicaftor (diponecaftor in registry); CTX-4430; PTI-428Clinical trial · NCT05668741Clinical trialTrial 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
VX-522 mRNA therapy with ivacaftor; dirocaftor/posenacaftor/nesolicaftor (diponecaftor in registry); CTX-4430; PTI-428Clinical trial · NCT05668741Clinical trialTrial 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 therapyClinical trial · NCT05699148Clinical trialTrial 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
Elexacaftor-tezacaftor-ivacaftor / Kaftrio-Trikafta; initiation of CFTR modulator therapyClinical trial · NCT05699148Clinical trialTrial 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

A clinical-trial listing or early report shows an option is being studied — not that it works, that it is safe for any one person, or that a site is enrolling today. Whether any of these fits is a conversation for your oncology team and the trial team. Last checked 2026-06-12.