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선택지 정리됨

Phenylketonuria (PKU): 국가별 선택지

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선택지 정리됨희귀·유전 질환최종 확인 2026.06

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임상시험 검색어

Phenylketonuria (PKU) 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.

European Union

  • sapropterin dihydrochloride (Kuvan); pegvaliase (Palynziq); sepiapterin (Sephience)[1]EMA authorisedPAH deficiency; BH4-responsive and sepiapterin-responsive contexts; Sapropterin as PKU/BH4-deficiency therapy and UK age-limited access; pegvaliase after inadequate Phe control despite prior management; sepiapterin in responsive PKU. · EMA authorisation does not by itself establish member-state reimbursement. NICE applies to England/Wales-type NHS contexts and is age/continuation constrained. HAS and G-BA are national HTA/reimbursement or benefit-assessment sources, not clinical eligibility determinations for an individual. Confidence/conflicts: High for EMA/NICE/HAS/G-BA sourced jurisdiction claims; no direct France/Germany Sephience reimbursement source captured yet.
  • sapropterin dihydrochloride (Kuvan); pegvaliase (Palynziq); sepiapterin (Sephience)[1]EMA authorisedPAH deficiency; BH4-responsive and sepiapterin-responsive contexts; Sapropterin as PKU/BH4-deficiency therapy and UK age-limited access; pegvaliase after inadequate Phe control despite prior management; sepiapterin in responsive PKU. · EMA authorisation does not by itself establish member-state reimbursement. NICE applies to England/Wales-type NHS contexts and is age/continuation constrained. HAS and G-BA are national HTA/reimbursement or benefit-assessment sources, not clinical eligibility determinations for an individual. Confidence/conflicts: High for EMA/NICE/HAS/G-BA sourced jurisdiction claims; no direct France/Germany Sephience reimbursement source captured yet.

Japan

  • sapropterin dihydrochloride (Biopten / Kuvan context); sepiapterin (Sephience)[2]PMDA-approved (Japan)PAH deficiency; BH4-responsive and sepiapterin-responsive contexts; BH4-responsive PKU for sapropterin; adult and pediatric PKU for sepiapterin per manufacturer approval announcement. · These Japan claims are manufacturer/regulatory-announcement sourced rather than direct PMDA label text. Current reimbursement, product name, and package-insert details need direct PMDA/MHLW verification. Confidence/conflicts: Medium because direct PMDA/MHLW labels were not fetched; no conflict identified.
  • sapropterin dihydrochloride (Biopten / Kuvan context); sepiapterin (Sephience)[2]PMDA-approved (Japan)PAH deficiency; BH4-responsive and sepiapterin-responsive contexts; BH4-responsive PKU for sapropterin; adult and pediatric PKU for sepiapterin per manufacturer approval announcement. · These Japan claims are manufacturer/regulatory-announcement sourced rather than direct PMDA label text. Current reimbursement, product name, and package-insert details need direct PMDA/MHLW verification. Confidence/conflicts: Medium because direct PMDA/MHLW labels were not fetched; no conflict identified.

Australia

  • pegvaliase (Palynziq)[3]TGA-registered (Australia)PAH deficiency; not limited to BH4-responsive disease in the cited TGA pegvaliase indication; PKU age 16 years and older with inadequate blood phenylalanine control despite prior management in Australia. · The AusPAR verifies registration and indication but not public subsidy. The TGA document records Black Triangle Scheme conditions and advisory discussion emphasizing careful monitoring because of adverse events. Confidence/conflicts: High for Australian TGA-approved indication; reimbursement status remains source-pending. No conflict identified.
  • pegvaliase (Palynziq)[3]TGA-registered (Australia)PAH deficiency; not limited to BH4-responsive disease in the cited TGA pegvaliase indication; PKU age 16 years and older with inadequate blood phenylalanine control despite prior management in Australia. · The AusPAR verifies registration and indication but not public subsidy. The TGA document records Black Triangle Scheme conditions and advisory discussion emphasizing careful monitoring because of adverse events. Confidence/conflicts: High for Australian TGA-approved indication; reimbursement status remains source-pending. No conflict identified.
  • phenylalanine-restricted diet, special low-protein foods, PKU formula/medical nutrition support[4]Standard option (per Australian Government Department of Health, Disability and Ageing)PAH deficiency / PKU; DHPR deficiency and HPA also included in the Australian IEM form source; lifelong dietary/specialised-food support and newborn/ongoing PKU nutrition management in Australia. · The IEM program is financial support for specialised food, not a drug approval. Reapplication intervals and metabolic-specialist involvement apply; individual nutrition targets are outside this catalog entry. Confidence/conflicts: High for Australian dietary-support pathway and PKU nutrition framing; no source conflict identified.
  • sepiapterin (Sephience)[5]TGA-registered (Australia)PAH deficiency; TGA registration page does not restrict to a named genotype; adult and paediatric PKU with HPA in Australia. · Registration and risk-management conditions are verified, but PBS subsidy or individual access criteria were not established. The advisory committee proposed wording included proven responsiveness, while the final TGA indication shown in the source is broader. Confidence/conflicts: High for Australian registration; medium for payer/access pathway, which is not established by these sources. Advisory wording and final indication differ in specificity, recorded above. Availability/reimbursement outside the approving regulator not established.

Thailand

  • sapropterin / tetrahydrobiopterin (BH4)[6]EMA authorisedelevated phenylalanine; PAH mutation, normal pterins/DHPR, or BH4 loading test criteria as listed in Thai access form; Thai national-list controlled-use/preauthorization context for sapropterin in PKU after dietary management and BH4 responsiveness criteria. · This is Thai-language access/form content and requires human review before patient-facing reuse. It is not a full product label and does not establish that every PKU patient is eligible; it documents a controlled-use pathway and criteria fields used for approval/continuation. Confidence/conflicts: Medium-high for existence of a Thai controlled-use pathway and form criteria; no conflict identified, but Thai-language clinical/access content needs human review and product-label details remain a gap. Primary source is in Thai. English summary pending human review — confirm exact wording with your care team. Availability/reimbursement outside the approving regulator not established.

Canada

  • pegvaliase (Palynziq)[7]Health Canada approvedPAH deficiency; inadequate phenylalanine control context; PKU age 16 years and older with blood Phe greater than 600 micromol/L despite dietary management in Canada. · The product monograph includes systemic hypersensitivity/anaphylaxis warnings and supervised initial administration requirements. CADTH reimbursement recommendation is conditional and does not guarantee provincial listing or individual access. Confidence/conflicts: High for Canadian label and CADTH conditional reimbursement recommendation; provincial implementation remains source-pending. No conflict identified.
  • pegvaliase (Palynziq)[7]Health Canada approvedPAH deficiency; inadequate phenylalanine control context; PKU age 16 years and older with blood Phe greater than 600 micromol/L despite dietary management in Canada. · The product monograph includes systemic hypersensitivity/anaphylaxis warnings and supervised initial administration requirements. CADTH reimbursement recommendation is conditional and does not guarantee provincial listing or individual access. Confidence/conflicts: High for Canadian label and CADTH conditional reimbursement recommendation; provincial implementation remains source-pending. No conflict identified.
  • sapropterin dihydrochloride (Kuvan)[8]Health Canada approvedPAH deficiency with BH4-responsive phenotype; BH4-responsive PKU with Phe-restricted diet in Canada. · Health Canada label does not establish provincial reimbursement or metabolic-clinic response criteria. The monograph states treatment should be directed by physicians knowledgeable in PKU management and that blood Phe levels should be monitored. Confidence/conflicts: High for Canadian label and marketed status; reimbursement status remains source-pending. No conflict identified.
  • sepiapterin for oral suspension (Sephience)[9]Health Canada approvedPAH deficiency; sepiapterin-responsive phenotype; sepiapterin-responsive PKU in patients 1 month and older in Canada, with Phe-restricted diet. · Health Canada notes long-term safety data are limited, especially for young pediatric patients, and that blood Phe levels and dietary Phe intake should be monitored. Reimbursement review/implementation was not established by the fetched Health Canada sources. Confidence/conflicts: High for Canadian approval, indication, and marketed status; reimbursement remains source-pending. No conflict identified.

출처

  1. European Medicines Agency (EMA) — regulator EPAR · regulator EPAR
  2. BioMarin Pharmaceutical — manufacturer regulatory approval announcement · manufacturer regulatory approval announcement
  3. Therapeutic Goods Administration (TGA) — Australian Public Assessment Report · Australian Public Assessment Report
  4. Australian Government Department of Health, Disability and Ageing — public health support program page · public health support program page
  5. Therapeutic Goods Administration (TGA) — prescription medicine registration page · prescription medicine registration page
  6. Thai National Drug Information / Thai FDA-MOPH — national-list controlled-use notice · national-list controlled-use notice
  7. Health Canada Drug Product Database — drug product database record · drug product database record
  8. Health Canada Drug Product Database — drug product database record · drug product database record
  9. Health Canada Drug Product Database — drug product database record · drug product database record

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.