RG 7800

Drug Profile

RG 7800

Alternative Names: RG 7800; RO6885247; SMN2 gene-targeted therapy - PTC Therapeutics; SMN2-targeted therapeutic for spinal muscular atrophy - PTC Therapeutics/SMA Foundation/Roche

Latest Information Update: 10 Sep 2017

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At a glance

  • Originator PTC Therapeutics
  • Developer Roche
  • Class Small molecules
  • Mechanism of Action Survival of motor neuron 1 protein stimulants; Survival of motor neuron 2 protein modulators
  • 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

  • Phase I/II Spinal muscular atrophy

Most Recent Events

  • 01 Jul 2016 Roche terminates the phase I/II MOONFISH trial in Spinal muscular atrophy (In children, In adolescents, In adults, In infants) in United Kingdom, Italy, Turkey, Switzerland, Canada, France, the Netherlands, Sweden and USA (NCT02240355)
  • 04 Oct 2015 Interim efficacy and adverse events data from a phase-I/II trial in Spinal muscular atrophy released by PTC Therapeutics
  • 19 Nov 2014 Phase-I/II clinical trials in Spinal muscular atrophy (In infants, In children, In adolescents, In adults) in Turkey, Italy and Switzerland (PO) (NCT02240355; EudraCT2014-002246-41)
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