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JR-141 or Idursulfase

JCR Pharmaceuticals Co., Ltd. · Phase 3 active Small molecule

JR-141 or Idursulfase is a Enzyme replacement therapy Small molecule drug developed by JCR Pharmaceuticals Co., Ltd.. It is currently in Phase 3 development for Hunter syndrome (mucopolysaccharidosis II, MPS II) with central nervous system involvement.

JR-141 is a recombinant idursulfase enzyme that replaces deficient iduronate-2-sulfatase to break down glycosaminoglycans in Hunter syndrome.

JR-141 is a recombinant idursulfase enzyme that replaces deficient iduronate-2-sulfatase to break down glycosaminoglycans in Hunter syndrome. Used for Hunter syndrome (mucopolysaccharidosis II, MPS II) with central nervous system involvement.

At a glance

Generic nameJR-141 or Idursulfase
SponsorJCR Pharmaceuticals Co., Ltd.
Drug classEnzyme replacement therapy
TargetIduronate-2-sulfatase
ModalitySmall molecule
Therapeutic areaRare genetic disease / Lysosomal storage disorder
PhasePhase 3

Mechanism of action

Hunter syndrome (mucopolysaccharidosis II) is caused by deficiency of the iduronate-2-sulfatase enzyme, leading to accumulation of glycosaminoglycans in tissues. JR-141 is an enzyme replacement therapy that provides the missing enzyme, enabling degradation of accumulated substrates. The drug is formulated to cross the blood-brain barrier and address both systemic and central nervous system manifestations of the disease.

Approved indications

Common side effects

Key clinical trials

Primary sources

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SourceUsed for
ClinicalTrials.govTrial enrolment, design, endpoints, results

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Frequently asked questions about JR-141 or Idursulfase

What is JR-141 or Idursulfase?

JR-141 or Idursulfase is a Enzyme replacement therapy drug developed by JCR Pharmaceuticals Co., Ltd., indicated for Hunter syndrome (mucopolysaccharidosis II, MPS II) with central nervous system involvement.

How does JR-141 or Idursulfase work?

JR-141 is a recombinant idursulfase enzyme that replaces deficient iduronate-2-sulfatase to break down glycosaminoglycans in Hunter syndrome.

What is JR-141 or Idursulfase used for?

JR-141 or Idursulfase is indicated for Hunter syndrome (mucopolysaccharidosis II, MPS II) with central nervous system involvement.

Who makes JR-141 or Idursulfase?

JR-141 or Idursulfase is developed by JCR Pharmaceuticals Co., Ltd. (see full JCR Pharmaceuticals Co., Ltd. pipeline at /company/jcr-pharmaceuticals-co-ltd).

What drug class is JR-141 or Idursulfase in?

JR-141 or Idursulfase belongs to the Enzyme replacement therapy class. See all Enzyme replacement therapy drugs at /class/enzyme-replacement-therapy.

What development phase is JR-141 or Idursulfase in?

JR-141 or Idursulfase is in Phase 3.

What are the side effects of JR-141 or Idursulfase?

Common side effects of JR-141 or Idursulfase include Infusion-related reactions, Antibody formation, Headache.

What does JR-141 or Idursulfase target?

JR-141 or Idursulfase targets Iduronate-2-sulfatase and is a Enzyme replacement therapy.

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