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Olipudase alfa - Sanofi Genzyme

Drug Profile

Olipudase alfa - Sanofi Genzyme

Alternative Names: GZ-402665; Recombinant human acid sphingomyelinase - Sanofi Genzyme; rhASM - Sanofi Genzyme

Latest Information Update: 04 Aug 2021

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At a glance

  • Originator Genzyme Corporation
  • Developer Genzyme Corporation; Sanofi Genzyme
  • Class Sphingomyelin phosphodiesterases
  • Mechanism of Action Sphingomyelin-phosphodiesterase replacements
  • Orphan Drug Status

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

    Yes - Niemann-Pick diseases
  • New Molecular Entity Yes

Highest Development Phases

  • Phase II/III Niemann-Pick disease type B
  • Clinical Phase Unknown Niemann-Pick diseases
  • No development reported Niemann-Pick disease type A

Most Recent Events

  • 09 Jun 2021 Efficacy and adverse events data from a phase I/II ASCEND-Peds trial for Niemann-Pick disease type B presented at the 26th Congress of the European Haematology Association (EHA-2021)
  • 07 May 2021 Sanofi initiates an Expanded access study (Compassionate Use Programme) for Niemann-Pick diseases (In children, In adolescents, In adults, In the elderly) (IV) (NCT04877132)
  • 23 Mar 2021 Clinical trials in Niemann-Pick diseases (compassionate Use) (In children, In adolescents, In adults, In the elderly) (IV) (NCT04799522)
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