Research programme: Niemann-Pick type A disease gene therapy - Sanofi Genzyme

Drug Profile

Research programme: Niemann-Pick type A disease gene therapy - Sanofi Genzyme

Alternative Names: Sphingomyelinase gene therapy

Latest Information Update: 16 Dec 2016

Price : $50

At a glance

  • Originator Genzyme Corporation
  • Class Gene therapies
  • Mechanism of Action Sphingomyelin phosphodiesterase stimulants
  • Orphan Drug Status

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

    No
  • On Fast track

    Fast track status is assigned by the US FDA so therapies with the potential to address unmet needs can move faster through development.

    No
  • New Molecular Entity Yes

Highest Development Phases

  • Discontinued Niemann-Pick disease type A

Most Recent Events

  • 26 Feb 2009 Discontinued - Preclinical for Niemann-Pick disease type A in USA (Injection)
  • 01 May 2006 This programme is still in active development
  • 22 Jun 2005 Data presented at the 8th Annual Meeting of the American Society of Gene Therapy (ASGT-2005) have been added to the metabolic disorders pharmacodynamics section
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