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Risdiplam - PTC Therapeutics/Roche

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Drug Profile

Risdiplam - PTC Therapeutics/Roche

Alternative Names: Evrysdi; EvrysdiTM; RG-7916; RO-7034067

Latest Information Update: 20 Jun 2024

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At a glance

  • Originator PTC Therapeutics
  • Developer Chugai Pharmaceutical; PTC Therapeutics; Roche
  • Class 2 ring heterocyclic compounds; Imidazoles; Pyridazines; Pyrimidines; Small molecules; Spiro compounds
  • Mechanism of Action 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.

    Yes - Spinal muscular atrophy
  • New Molecular Entity Yes

Highest Development Phases

  • Marketed Spinal muscular atrophy

Most Recent Events

  • 07 Jun 2024 Efficacy and adverse events data from phase II FIREFISH trial in Spinal muscular atrophy released by Roche
  • 26 Apr 2024 Hoffmann-La Roche initiates a phase II trial for Spinal muscular atrophy in the US, Belgium and Germany (PO) (NCT05808764)
  • 13 Apr 2024 Efficacy and adverse events data from phase II RAINBOWFISH trial in Spinal muscular atrophy were presented at 76th Annual Meeting of the American Academy of Neurology 2024 (AAN-2024)

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