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Mucopolysaccharidosis (MPS): options by country

Sourced options by country plus visit-prep questions for Mucopolysaccharidosis (MPS). 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 MPS subtype is confirmed by enzyme and/or genetic testing?
  • Are the symptoms primarily systemic/non-CNS, neurologic/CNS, airway, cardiac, skeletal, or mixed?
  • Is enzyme replacement being discussed alone, around transplant, or as part of supportive multidisciplinary care?
  • Which MPS subtype and enzyme deficiency are documented?

Trial-search terms to discuss

Mucopolysaccharidosis (MPS) 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

  • Laronidase (Aldurazyme); idursulfase (Elaprase)[1]FDA-approvedAlpha-L-iduronidase deficiency / IDUA mutation for MPS I; iduronate-2-sulfatase deficiency / IDS mutation for MPS II; Long-term enzyme replacement therapy for non-CNS/systemic MPS I or MPS II manifestations as described in labels. · Aldurazyme has not been evaluated for central nervous system manifestations. Elaprase label includes age/symptom caveats and infusion/anaphylaxis precautions. Hematopoietic stem-cell transplant and supportive care are not fully captured in this first pass. Confidence/conflicts: High for U.S. FDA-label indications; CNS/transplant/supportive pathways need deeper source work.
  • Laronidase (Aldurazyme); idursulfase (Elaprase)[1]FDA-approvedAlpha-L-iduronidase deficiency / IDUA mutation for MPS I; iduronate-2-sulfatase deficiency / IDS mutation for MPS II; Long-term enzyme replacement therapy for non-CNS/systemic MPS I or MPS II manifestations as described in labels. · Aldurazyme has not been evaluated for central nervous system manifestations. Elaprase label includes age/symptom caveats and infusion/anaphylaxis precautions. Hematopoietic stem-cell transplant and supportive care are not fully captured in this first pass. Confidence/conflicts: High for U.S. FDA-label indications; CNS/transplant/supportive pathways need deeper source work.
  • Laronidase (Aldurazyme); idursulfase (Elaprase)[1]FDA-approvedAlpha-L-iduronidase deficiency / IDUA mutation for MPS I; iduronate-2-sulfatase deficiency / IDS mutation for MPS II; Long-term enzyme replacement therapy for non-CNS/systemic MPS I or MPS II manifestations as described in labels. · Aldurazyme has not been evaluated for central nervous system manifestations. Elaprase label includes age/symptom caveats and infusion/anaphylaxis precautions. Hematopoietic stem-cell transplant and supportive care are not fully captured in this first pass. Confidence/conflicts: High for U.S. FDA-label indications; CNS/transplant/supportive pathways need deeper source work.

European Union

  • Aldurazyme; Elaprase; Vimizim; Mepsevii[2]EMA authorisedAlpha-L-iduronidase deficiency; iduronate-2-sulfatase deficiency; GALNS deficiency for MPS IVA; beta-glucuronidase deficiency for MPS VII; Long-term ERT by MPS subtype; MPS I source explicitly non-neurological manifestations. · EMA authorization does not equal member-state reimbursement. UK, France, and Germany access/reimbursement remain source-pending. MPS VII and MPS IVA entries are adjacent to Hurler/Hunter but included to avoid missing available MPS-category options. Confidence/conflicts: High for EMA authorizations; medium for UK detail pending final NICE subtype-by-subtype pass.
  • Aldurazyme; Elaprase; Vimizim; Mepsevii[2]EMA authorisedAlpha-L-iduronidase deficiency; iduronate-2-sulfatase deficiency; GALNS deficiency for MPS IVA; beta-glucuronidase deficiency for MPS VII; Long-term ERT by MPS subtype; MPS I source explicitly non-neurological manifestations. · EMA authorization does not equal member-state reimbursement. UK, France, and Germany access/reimbursement remain source-pending. MPS VII and MPS IVA entries are adjacent to Hurler/Hunter but included to avoid missing available MPS-category options. Confidence/conflicts: High for EMA authorizations; medium for UK detail pending final NICE subtype-by-subtype pass.

Japan

  • elosulfase alfa (Vimizim)[3]PMDA-approved (Japan)N-acetylgalactosamine-6-sulfatase / GALNS deficiency context; source names MPS IVA; enzyme replacement therapy for MPS IVA / Morquio A syndrome. · The fetched PMDA list verifies approval and indication but does not provide full label details, reimbursement, respiratory/orthopedic/anesthesia precautions, infusion-reaction management, or mobility endpoint limitations. Full current Japanese package insert remains a gap. Confidence/conflicts: High for Japanese Vimizim MPS IVA approval and indication from PMDA list; no conflict identified. Full label and reimbursement remain gaps.
  • idursulfase (Elaprase)[3]PMDA-approved (Japan)iduronate-2-sulfatase (IDS) deficiency context; X-linked recessive MPS II; intravenous enzyme replacement therapy for mucopolysaccharidosis II. · PMDA's Izcargo review report states idursulfase/Elaprase is currently available in Japan as intravenous enzyme replacement therapy for MPS II, but because IDS cannot cross the blood-brain barrier, intravenous Elaprase is unlikely to treat CNS symptoms. This entry does not establish reimbursement, long-term neurologic outcomes, infusion reaction protocols, or full current package insert details. Confidence/conflicts: High for Japanese Elaprase MPS II approval and CNS limitation context; no conflict identified. Full current label and reimbursement remain gaps.
  • pabinafusp alfa (Izcargo for I.V. Infusion)[4]PMDA-approved (Japan)iduronate-2-sulfatase (IDS) deficiency context; CNS symptoms/progression context emphasized in source; weekly intravenous treatment for MPS II; PMDA accepted a precaution that pabinafusp alfa should be administered to patients in whom improvement of CNS symptoms or suppression of CNS-symptom progression is considered necessary. · PMDA states long-term efficacy and other aspects should be further evaluated. The review notes antibody production and recommends regular anti-drug antibody testing, with close monitoring for infusion-associated reactions in antibody-positive patients. This entry does not establish reimbursement, comparative choice versus Elaprase, or individual eligibility. Confidence/conflicts: High for Japanese Izcargo MPS II approval and CNS-precaution context; no conflict identified. Long-term evidence and reimbursement remain gaps.

Australia

  • idursulfase (Elaprase)[5]Approvediduronate-2-sulfatase deficiency / IDS mutation context; Australian LSDP access for idursulfase in MPS II; long-term ERT for Hunter syndrome. · LSDP access is subject to guideline eligibility and reapplication requirements. Healthdirect reflects ARTG medicine information but is not a full TGA PI or payer rule. Confidence/conflicts: Medium-high; Australian access and ARTG-linked indication verified, but full current PI criteria remain source-pending. Availability/reimbursement outside the approving regulator not established.
  • idursulfase (Elaprase)[5]Approvediduronate-2-sulfatase deficiency / IDS mutation context; Australian LSDP access for idursulfase in MPS II; long-term ERT for Hunter syndrome. · LSDP access is subject to guideline eligibility and reapplication requirements. Healthdirect reflects ARTG medicine information but is not a full TGA PI or payer rule. Confidence/conflicts: Medium-high; Australian access and ARTG-linked indication verified, but full current PI criteria remain source-pending. Availability/reimbursement outside the approving regulator not established.
  • laronidase (Aldurazyme); idursulfase (Elaprase)[6]ApprovedMPS I alpha-L-iduronidase deficiency; MPS II iduronate-2-sulfatase deficiency; Australian LSDP-funded ERT access pathways for MPS I and MPS II. · The general LSDP page identifies funded medicines and conditions but not full clinical criteria. Subtype-specific guidelines and current PI should be checked before reuse. Confidence/conflicts: Medium-high for Australian LSDP medicine-condition mapping; detailed MPS I guideline extraction remains a follow-up. Availability/reimbursement outside the approving regulator not established.
  • laronidase (Aldurazyme); idursulfase (Elaprase)[6]ApprovedMPS I alpha-L-iduronidase deficiency; MPS II iduronate-2-sulfatase deficiency; Australian LSDP-funded ERT access pathways for MPS I and MPS II. · The general LSDP page identifies funded medicines and conditions but not full clinical criteria. Subtype-specific guidelines and current PI should be checked before reuse. Confidence/conflicts: Medium-high for Australian LSDP medicine-condition mapping; detailed MPS I guideline extraction remains a follow-up. Availability/reimbursement outside the approving regulator not established.

Thailand

  • idursulfase (Elaprase)[7]Approvediduronate-2-sulfatase deficiency / IDS-related MPS II context; severe mutation status relevant for hypersensitivity/antibody risk per source; enzyme replacement therapy for Hunter syndrome/MPS II; source specifies evidence distinctions by age group. · Safety and efficacy are not established in pediatric patients younger than 16 months. The label includes serious hypersensitivity/anaphylaxis, respiratory/cardiac monitoring, and antibody-risk cautions, including higher risks in patients with complete gene deletion, large rearrangement, nonsense, frameshift, or splice-site mutations. This entry does not establish reimbursement or center availability in Thailand. Confidence/conflicts: High for Thai Elaprase label and authorization details; no conflict identified, but reimbursement/access pathway remains unresolved.

Canada

  • idursulfase (Elaprase)[8]Health Canada approvediduronate-2-sulfatase deficiency; long-term ERT for MPS II/Hunter syndrome in Canada. · The product monograph verifies label status but not public-plan coverage. It notes supplemental oxygen should be available during infusion for patients who use oxygen if an infusion-related reaction occurs. Confidence/conflicts: High for Canadian Elaprase label and marketed status; no conflict identified. Availability/reimbursement outside the approving regulator not established.
  • idursulfase (Elaprase)[8]Health Canada approvediduronate-2-sulfatase deficiency; long-term ERT for MPS II/Hunter syndrome in Canada. · The product monograph verifies label status but not public-plan coverage. It notes supplemental oxygen should be available during infusion for patients who use oxygen if an infusion-related reaction occurs. Confidence/conflicts: High for Canadian Elaprase label and marketed status; no conflict identified. Availability/reimbursement outside the approving regulator not established.
  • laronidase (Aldurazyme)[9]Approvedalpha-L-iduronidase deficiency; long-term ERT for non-CNS manifestations of MPS I in Canada. · Aldurazyme does not address central nervous system manifestations in the label wording. The monograph highlights life-threatening anaphylactic reactions and need for appropriate medical support during and after infusion. Confidence/conflicts: High for Canadian label; reimbursement/HSCT pathway remains source-pending.
  • laronidase (Aldurazyme)[9]Approvedalpha-L-iduronidase deficiency; long-term ERT for non-CNS manifestations of MPS I in Canada. · Aldurazyme does not address central nervous system manifestations in the label wording. The monograph highlights life-threatening anaphylactic reactions and need for appropriate medical support during and after infusion. Confidence/conflicts: High for Canadian label; reimbursement/HSCT pathway remains source-pending.

Sources

  1. U.S. Food and Drug Administration — official drug label · official drug label
  2. European Medicines Agency (EMA) — regulator EPAR · regulator EPAR
  3. Pharmaceuticals and Medical Devices Agency (PMDA) — approved drugs list PDF · approved drugs list PDF
  4. Pharmaceuticals and Medical Devices Agency (PMDA) — regulator deliberation/review report PDF · regulator deliberation/review report PDF
  5. Australian Government Department of Health, Disability and Ageing — LSDP resource collection · LSDP resource collection
  6. Australian Government Department of Health, Disability and Ageing — LSDP program description · LSDP program description
  7. Thai National Drug Information / Thai FDA-MOPH — SmPC PDF / regulator drug-information repository · SmPC PDF / regulator drug-information repository
  8. Health Canada product monograph repository / Takeda Canada — official product monograph · official product monograph
  9. Sanofi-aventis Canada / Health Canada product monograph — official product monograph · official product monograph

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

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.