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/RocheLatest Information Update: 31 Jul 2018
Price :
$50
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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
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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
- Discontinued 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)