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Inherited retinal dystrophy (RPE65): options by country

Sourced options by country plus visit-prep questions for Inherited retinal dystrophy (RPE65). 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

  • Has genetic testing confirmed biallelic RPE65 mutations, and is there enough viable retina in the eye being considered?
  • Which certified/experienced retinal gene-therapy center can evaluate administration and surgical risks?
  • What monitoring is needed for inflammation, retinal changes, cataract, intraocular pressure, and travel restrictions after treatment?
  • Is the question EMA authorization, England NICE access, Scotland/Wales/Northern Ireland policy, or France/Germany reimbursement?

Trial-search terms to discuss

Inherited retinal dystrophy (RPE65) 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

  • voretigene neparvovec-rzyl (Luxturna)[1]FDA-approvedconfirmed biallelic RPE65 mutations; viable retinal cells required in label/source context; One-time subretinal gene therapy for each eligible eye in confirmed biallelic RPE65 mutation-associated retinal dystrophy, if viable retinal cells are present. · Genetic confirmation and sufficient viable retinal cells are central source caveats. The label includes surgical-suite administration, corticosteroid regimen, intraocular-injection risks, retinal abnormalities, intraocular pressure, air-bubble precautions, cataract, and pediatric-use caveats. This entry does not imply eligibility or availability at every eye center. Confidence/conflicts: High for U.S. label-backed status; no conflicts identified.
  • voretigene neparvovec-rzyl (Luxturna)[1]FDA-approvedconfirmed biallelic RPE65 mutations; viable retinal cells required in label/source context; One-time subretinal gene therapy for each eligible eye in confirmed biallelic RPE65 mutation-associated retinal dystrophy, if viable retinal cells are present. · Genetic confirmation and sufficient viable retinal cells are central source caveats. The label includes surgical-suite administration, corticosteroid regimen, intraocular-injection risks, retinal abnormalities, intraocular pressure, air-bubble precautions, cataract, and pediatric-use caveats. This entry does not imply eligibility or availability at every eye center. Confidence/conflicts: High for U.S. label-backed status; no conflicts identified.

European Union

  • voretigene neparvovec (Luxturna)[2]EMA authorisedconfirmed biallelic RPE65 mutations; sufficient viable retinal cells; Gene therapy for RPE65-mediated inherited retinal dystrophy with vision loss and viable retinal cells. · EMA authorization does not by itself establish reimbursement in each EU member state. NICE guidance is UK/England health-technology guidance and lists its next review as 2022, so current access details should be refreshed with local commissioning sources. Confidence/conflicts: High for EMA/NICE scope; medium for current UK operational access because refreshed commissioning details were not fetched this cycle.
  • voretigene neparvovec (Luxturna)[2]EMA authorisedconfirmed biallelic RPE65 mutations; sufficient viable retinal cells; Gene therapy for RPE65-mediated inherited retinal dystrophy with vision loss and viable retinal cells. · EMA authorization does not by itself establish reimbursement in each EU member state. NICE guidance is UK/England health-technology guidance and lists its next review as 2022, so current access details should be refreshed with local commissioning sources. Confidence/conflicts: High for EMA/NICE scope; medium for current UK operational access because refreshed commissioning details were not fetched this cycle.
  • voretigene neparvovec (Luxturna)[3]EMA authorisedconfirmed biallelic RPE65 mutations; sufficient viable retinal cells; Reimbursement opinion for Luxturna in the stated RPE65-mediated inherited retinal dystrophy population. · HAS favourable reimbursement opinion does not by itself identify every treating center, individual eligibility outcome, or administrative access step. Long-term follow-up and chorioretinal atrophy uncertainty are noted in the HAS summary. Confidence/conflicts: High for France reimbursement-opinion status; no conflict identified.
  • voretigene neparvovec (Luxturna)[3]EMA authorisedconfirmed biallelic RPE65 mutations; sufficient viable retinal cells; Reimbursement opinion for Luxturna in the stated RPE65-mediated inherited retinal dystrophy population. · HAS favourable reimbursement opinion does not by itself identify every treating center, individual eligibility outcome, or administrative access step. Long-term follow-up and chorioretinal atrophy uncertainty are noted in the HAS summary. Confidence/conflicts: High for France reimbursement-opinion status; no conflict identified.
  • voretigene neparvovec (Luxturna)[4]Approvedconfirmed biallelic RPE65 mutations; sufficient viable retinal cells; German benefit assessment/quality-assured use context for Luxturna in the approved RPE65-mediated inherited retinal dystrophy population. · The English G-BA documents are courtesy translations and the German versions are legally binding. Earlier resolution validity was time-limited; this entry records the fetched G-BA benefit-assessment and resolution context, not a fresh 2026 reimbursement contract. Confidence/conflicts: Medium-high for German benefit-assessment context; medium for current access because the fetched English resolution is time-limited and legally non-binding.
  • voretigene neparvovec (Luxturna)[4]Approvedconfirmed biallelic RPE65 mutations; sufficient viable retinal cells; German benefit assessment/quality-assured use context for Luxturna in the approved RPE65-mediated inherited retinal dystrophy population. · The English G-BA documents are courtesy translations and the German versions are legally binding. Earlier resolution validity was time-limited; this entry records the fetched G-BA benefit-assessment and resolution context, not a fresh 2026 reimbursement contract. Confidence/conflicts: Medium-high for German benefit-assessment context; medium for current access because the fetched English resolution is time-limited and legally non-binding.

Japan

  • voretigene neparvovec (Luxturna Injection)[5]PMDA-approved (Japan)biallelic RPE65 mutation confirmed by genetic testing; sufficient viable retinal cells per PMDA review; Gene therapy for biallelic RPE65 mutation-associated inherited retinal dystrophy; Japan review includes LCA/RP phenotype discussion but supports gene-based indication wording. · PMDA review notes limited information for some phenotype subgroups and emphasizes gene-based selection plus retinal viability. Reimbursement and treatment-center logistics were not fully verified in this pass. Confidence/conflicts: High for PMDA approval/review status; no conflict identified.
  • voretigene neparvovec (Luxturna Injection)[5]PMDA-approved (Japan)biallelic RPE65 mutation confirmed by genetic testing; sufficient viable retinal cells per PMDA review; Gene therapy for biallelic RPE65 mutation-associated inherited retinal dystrophy; Japan review includes LCA/RP phenotype discussion but supports gene-based indication wording. · PMDA review notes limited information for some phenotype subgroups and emphasizes gene-based selection plus retinal viability. Reimbursement and treatment-center logistics were not fully verified in this pass. Confidence/conflicts: High for PMDA approval/review status; no conflict identified.
  • voretigene neparvovec (Luxturna Injection)[6]PMDA-approved (Japan)confirmed biallelic RPE65 gene mutations; viable retinal-cell assessment is relevant per source context; inherited retinal dystrophy caused by biallelic RPE65 gene mutations; subretinal administration as a gene therapy product. · The PMDA review describes Luxturna as a recombinant adeno-associated virus vector carrying the human RPE65 gene and expected to improve visual function by compensating for loss of RPE65 function. This entry does not establish Japanese reimbursement details, designated treatment-center requirements, genetic-testing access, or individual eligibility; specialist ophthalmology/genetics evaluation is required. Confidence/conflicts: High for Japanese PMDA approval and indication summary; no conflict identified. Reimbursement, center designation, and testing pathway remain unresolved.

Australia

  • ophthalmic assessment, genetic testing/counselling, low-vision and mobility support, gene-therapy referral where relevant[7]Standard option (per Retina Australia)over 300 IRD-associated genes; RPE65 is one gene-specific therapy context; Australian genetic testing/counselling, registry, and supportive-care infrastructure for IRDs before or alongside gene-specific treatment. · These are patient-support/education sources, not gene-therapy eligibility rules. A negative or inconclusive genetic test does not rule out IRD, and RPE65 therapy only applies to the specific biallelic RPE65 context with viable retinal cells. Confidence/conflicts: Medium-high for Australian support/genetic-testing navigation; not a regulator or payer source. No conflict identified.
  • specialist IRD assessment and management guideline; genetic diagnosis and gene-therapy readiness context[8]Standard option (per Royal Australian and New Zealand College of Ophthalmologists)gene-specific diagnosis required for gene-targeted therapy; RPE65 and many non-RPE65 genes; Professional ophthalmology assessment/management framework for inherited retinal degenerations, including gene-therapy-era considerations. · This entry records professional-guideline context and does not extract a drug-specific recommendation beyond the document's scope and references. Specific treatment eligibility must be checked against current TGA/MSAC/Luxturna criteria and specialist center protocols. Confidence/conflicts: Medium-high for professional-guideline context; current specific gene-therapy recommendations need live source confirmation. No conflict identified.
  • voretigene neparvovec (Luxturna)[9]TGA-registered (Australia)pathological biallelic RPE65 mutations confirmed by NATA or ILAC accredited laboratory; sufficient viable retinal cells; Gene therapy for biallelic RPE65-mediated inherited retinal dystrophy; service/technology delivery requires subretinal injection after vitrectomy per MSAC summary. · TGA approval is not the same as full funding/access at every Australian site. MSAC source documents assessment context but this entry does not map current state-by-state treatment-center logistics. Confidence/conflicts: High for TGA approval and MSAC assessment context; medium for current funding/treatment-center pathways. confirm current TGA ARTG registration status Availability/reimbursement outside the approving regulator not established.
  • voretigene neparvovec (Luxturna)[9]TGA-registered (Australia)pathological biallelic RPE65 mutations confirmed by NATA or ILAC accredited laboratory; sufficient viable retinal cells; Gene therapy for biallelic RPE65-mediated inherited retinal dystrophy; service/technology delivery requires subretinal injection after vitrectomy per MSAC summary. · TGA approval is not the same as full funding/access at every Australian site. MSAC source documents assessment context but this entry does not map current state-by-state treatment-center logistics. Confidence/conflicts: High for TGA approval and MSAC assessment context; medium for current funding/treatment-center pathways. confirm current TGA ARTG registration status Availability/reimbursement outside the approving regulator not established.
  • voretigene neparvovec (Luxturna)[10]Approvedconfirmed biallelic RPE65 pathogenic variants; sufficient viable retinal cells; Australian public funding/service-delivery assessment context for Luxturna in RPE65-mediated IRD. · MSAC funding/service advice is not the same as individual eligibility or treatment-center availability. Registry/managed-access data collection and specialist center arrangements may apply. Confidence/conflicts: High for Australian MSAC funding-service assessment; treatment-center map remains source-pending. No conflict identified. Availability/reimbursement outside the approving regulator not established.
  • voretigene neparvovec (Luxturna)[10]Approvedconfirmed biallelic RPE65 pathogenic variants; sufficient viable retinal cells; Australian public funding/service-delivery assessment context for Luxturna in RPE65-mediated IRD. · MSAC funding/service advice is not the same as individual eligibility or treatment-center availability. Registry/managed-access data collection and specialist center arrangements may apply. Confidence/conflicts: High for Australian MSAC funding-service assessment; treatment-center map remains source-pending. No conflict identified. Availability/reimbursement outside the approving regulator not established.
  • voretigene neparvovec (Luxturna)[10]Approvedconfirmed biallelic RPE65 pathogenic variants; sufficient viable retinal cells; Australian public funding/service-delivery assessment context for Luxturna in RPE65-mediated IRD. · MSAC funding/service advice is not the same as individual eligibility or treatment-center availability. Registry/managed-access data collection and specialist center arrangements may apply. Confidence/conflicts: High for Australian MSAC funding-service assessment; treatment-center map remains source-pending. No conflict identified. Availability/reimbursement outside the approving regulator not established.

Canada

  • voretigene neparvovec (Luxturna)[11]Approvedconfirmed biallelic RPE65 mutations; sufficient viable retinal cells; Canadian Health Canada/CADTH review context for one-time subretinal gene therapy in RPE65-mediated IRD with viable retinal cells. · CADTH review is not province-specific reimbursement. Genetic confirmation, retinal viability, treatment-center readiness, surgery, immunomodulation, and follow-up all require specialist inherited-retinal-disease assessment. Confidence/conflicts: High for Canadian indication/review context; reimbursement varies by province. No conflict identified.
  • voretigene neparvovec (Luxturna)[11]Approvedconfirmed biallelic RPE65 mutations; sufficient viable retinal cells; Canadian Health Canada/CADTH review context for one-time subretinal gene therapy in RPE65-mediated IRD with viable retinal cells. · CADTH review is not province-specific reimbursement. Genetic confirmation, retinal viability, treatment-center readiness, surgery, immunomodulation, and follow-up all require specialist inherited-retinal-disease assessment. Confidence/conflicts: High for Canadian indication/review context; reimbursement varies by province. No conflict identified.
  • voretigene neparvovec (Luxturna)[12]Health Canada approvedconfirmed biallelic RPE65 mutations; sufficient viable retinal cells; BC PharmaCare coverage-decision context for Luxturna in RPE65-mediated IRD. · The decision document is informational and does not replace physician advice. Exact BC special authority/current coverage criteria should be checked directly before assuming eligibility. Confidence/conflicts: High for BC coverage-decision context; exact live criteria remain source-pending. No conflict identified.
  • voretigene neparvovec (Luxturna)[12]Health Canada approvedconfirmed biallelic RPE65 mutations; sufficient viable retinal cells; BC PharmaCare coverage-decision context for Luxturna in RPE65-mediated IRD. · The decision document is informational and does not replace physician advice. Exact BC special authority/current coverage criteria should be checked directly before assuming eligibility. Confidence/conflicts: High for BC coverage-decision context; exact live criteria remain source-pending. No conflict identified.
  • voretigene neparvovec (Luxturna)[12]Health Canada approvedconfirmed biallelic RPE65 mutations; sufficient viable retinal cells; BC PharmaCare coverage-decision context for Luxturna in RPE65-mediated IRD. · The decision document is informational and does not replace physician advice. Exact BC special authority/current coverage criteria should be checked directly before assuming eligibility. Confidence/conflicts: High for BC coverage-decision context; exact live criteria remain source-pending. No conflict identified.

Sources

  1. DailyMed / U.S. National Library of Medicine — official drug label · official drug label
  2. European Medicines Agency (EMA) — regulator EPAR · regulator EPAR
  3. Haute Autorité de Santé (HAS) — health technology reimbursement opinion · health technology reimbursement opinion
  4. Gemeinsamer Bundesausschuss (G-BA) — benefit-assessment justification · benefit-assessment justification
  5. Pharmaceuticals and Medical Devices Agency (PMDA) — regulator approved-products review list · regulator approved-products review list
  6. Pharmaceuticals and Medical Devices Agency (PMDA) — regulator review report PDF · regulator review report PDF
  7. Retina Australia — patient-advocacy education · patient-advocacy education
  8. Royal Australian and New Zealand College of Ophthalmologists (RANZCO) — professional guideline · professional guideline
  9. Therapeutic Goods Administration (TGA) — regulator public assessment summary · regulator public assessment summary
  10. Medical Services Advisory Committee (MSAC) — public summary document / funding-service assessment · public summary document / funding-service assessment
  11. NCBI Bookshelf / CADTH — clinical review report · clinical review report
  12. British Columbia Ministry of Health / PharmaCare — provincial drug coverage decision · provincial drug coverage 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

  • voretigene neparvovec-rzyl (Luxturna / AAV2-hRPE65v2); AAV OPTIRPE65; tgAAG76 (rAAV 2/2.hRPE65p.hRPE65)Clinical trial · NCT03597399Clinical trialTrial only (NCT03597399)United States · RPE65 mutation-associated inherited retinal disease; Post-authorization treated-patient registry, long-term follow-up, and early gene-therapy clinical studies. · Registry/follow-up studies are not substitutes for current coverage or reimbursement decisions. The CADTH source is a clinical review, not a live provincial reimbursement map. Direct Thailand, Russia, and South Korea regulator/reimbursement sources were not verified. Confidence/conflicts: High for registry geography/status and Canadian clinical-review indication; medium for current Canada reimbursement because live provincial coverage was not verified. ClinicalTrials.gov — post-authorization registry
  • voretigene neparvovec-rzyl (Luxturna / AAV2-hRPE65v2); AAV OPTIRPE65; tgAAG76 (rAAV 2/2.hRPE65p.hRPE65)Clinical trial · NCT03597399Clinical trialTrial only (NCT03597399)United States · RPE65 mutation-associated inherited retinal disease; Post-authorization treated-patient registry, long-term follow-up, and early gene-therapy clinical studies. · Registry/follow-up studies are not substitutes for current coverage or reimbursement decisions. The CADTH source is a clinical review, not a live provincial reimbursement map. Direct Thailand, Russia, and South Korea regulator/reimbursement sources were not verified. Confidence/conflicts: High for registry geography/status and Canadian clinical-review indication; medium for current Canada reimbursement because live provincial coverage was not verified. ClinicalTrials.gov — post-authorization registry
  • voretigene neparvovec-rzyl (Luxturna / AAV2-hRPE65v2); AAV OPTIRPE65; tgAAG76 (rAAV 2/2.hRPE65p.hRPE65)Clinical trial · NCT03597399Clinical trialTrial only (NCT03597399)United States · RPE65 mutation-associated inherited retinal disease; Post-authorization treated-patient registry, long-term follow-up, and early gene-therapy clinical studies. · Registry/follow-up studies are not substitutes for current coverage or reimbursement decisions. The CADTH source is a clinical review, not a live provincial reimbursement map. Direct Thailand, Russia, and South Korea regulator/reimbursement sources were not verified. Confidence/conflicts: High for registry geography/status and Canadian clinical-review indication; medium for current Canada reimbursement because live provincial coverage was not verified. ClinicalTrials.gov — post-authorization registry
  • HG004 investigational gene therapy; FT-001 investigational gene therapyClinical trial · NCT05906953Clinical trialTrial only (NCT05906953)China · RPE65 mutations; biallelic status depends on specific protocol/source; Investigational gene-therapy studies for RPE65-related inherited retinal disease or Leber congenital amaurosis; protocols determine eligibility. · These are investigational trial/IND records, not Luxturna approval in China. Routine Chinese approval, reimbursement, and availability of Luxturna were not verified this cycle. Confidence/conflicts: High for China/U.S. investigational trial status; low for routine China approved access because no direct approval source was verified. ClinicalTrials.gov — clinical-trial registry
  • HG004 investigational gene therapy; FT-001 investigational gene therapyClinical trial · NCT05906953Clinical trialTrial only (NCT05906953)China · RPE65 mutations; biallelic status depends on specific protocol/source; Investigational gene-therapy studies for RPE65-related inherited retinal disease or Leber congenital amaurosis; protocols determine eligibility. · These are investigational trial/IND records, not Luxturna approval in China. Routine Chinese approval, reimbursement, and availability of Luxturna were not verified this cycle. Confidence/conflicts: High for China/U.S. investigational trial status; low for routine China approved access because no direct approval source was verified. 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.