Research programme: spinal muscular atrophy therapeutics - deCODE genetics/Families of Spinal Muscular Atrophy

Drug Profile

Research programme: spinal muscular atrophy therapeutics - deCODE genetics/Families of Spinal Muscular Atrophy

Latest Information Update: 07 Apr 2015

Price : $50

At a glance

  • Originator deCODE genetics; Families of SMA
  • Class Piperidines; Quinazolines; Small molecules
  • Mechanism of Action Survival of motor neuron 1 protein stimulants
  • Orphan Drug Status

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

    Yes - Spinal muscular atrophy
  • 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 Spinal muscular atrophy

Most Recent Events

  • 28 Aug 2009 Quinazoline 495 receives Orphan Drug status for Spinal muscular atrophy in USA
  • 15 Aug 2008 Research programme is still in preclinical trials for Spinal muscular atrophy in USA and Iceland
  • 26 Jun 2007 A clinical candidate has been selected for spinal muscular atrophy
Restricted Access

Oops, it looks like you don’t have a valid subscription to this content. To gain full access to the content and functionality of the AdisInsight database try one of the following.

  • with a username/password associated to an account with a valid subscription
  • Contact your organization’s admin about adding this content to your AdisInsight subscription
  • Request a trial

If you are a subscriber to this content then contact us at AsktheExpert.AdisInsight@springer.com so we can help.

Back to top