Research programme: scavenger mRNA decapping enzyme inhibitors - FSMA/Repligen

Drug Profile

Research programme: scavenger mRNA decapping enzyme inhibitors - FSMA/Repligen

Alternative Names: D 157495; Spinal muscular atrophy (SMA) programme - Repligen/Pfizer

Latest Information Update: 24 Nov 2015

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At a glance

  • Originator Families of SMA
  • Developer Families of SMA; Repligen Corporation
  • Class Quinazolines
  • 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.

  • New Molecular Entity Yes

Highest Development Phases

  • Preclinical Spinal muscular atrophy

Most Recent Events

  • 15 Mar 2015 Repligen has patent protection for spinal muscular atrophy programme in USA (Repligen Form 10-K filed in March 2015)
  • 03 Jan 2013 Repligen's spinal muscular atrophy programme (including RG 3039 and backup compounds) sub-licensed to Pfizer worldwide
  • 24 May 2011 A lead candidate, RG 3039, enters into clinical development
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