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Pabinafusp alfa - JCR Pharmaceuticals

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Drug Profile

Pabinafusp alfa - JCR Pharmaceuticals

Alternative Names: IZCARGO; JR 141; Recombinant iduronate-2-sulfatase - JCR Pharmaceuticals; TAK-141

Latest Information Update: 08 Jul 2025

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At a glance

  • Originator JCR Pharmaceuticals
  • Class Antibodies; Iduronate sulfatases; Recombinant fusion proteins
  • Mechanism of Action Iduronate sulfatase replacements
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Mucopolysaccharidosis II
  • New Molecular Entity Yes

Highest Development Phases

  • Marketed Mucopolysaccharidosis II

Most Recent Events

  • 02 Jul 2025 JCR Pharmaceuticals completes enrolment in a phase III trial in Mucopolysaccharidosis II (In children, In adolescents, In adults, In the elderly) in USA, Argentina, Brazil, Coloumbia, Israel, Italy, Poland, Turkey, United Kingdom, Spain, Germany and France (IV) (NCT04573023)
  • 30 Jun 2024 Takeda and JCR enters into an agreement ending the geographically-focused exclusive collaboration and license agreement to commercialize pabinafusp alfa in Mucopolysaccharidosis-II (Takeda Pipeline, February 2025)
  • 06 Apr 2023 JCR Pharmaceuticals and Sumitomo Pharma agree to co-promote pabinafusp alfa in Japan for Mucopolysaccharidosis-II

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