Risdiplam
CAS 1825352-65-5 7 FDA-DMF suppliersSmall Molecule
Overview
Risdiplam is a small molecule designed to increase the production of the survival motor neuron (SMN) protein, thereby treating spinal muscular atrophy (SMA). It is an oral medication that modulates pre-mRNA splicing of the SMN2 gene to promote the inclusion of exon 7 during mRNA processing.
Patent & exclusivity (US)
Per the FDA Orange Book (2 approved US products), the latest listed US patent on Risdiplam expires 14 Apr 2041 and exclusivity 26 May 2029. Generic API sourcing typically opens up once these lapse.
Therapeutic areas
Neurodegenerative DiseasesMusculoskeletal Diseases
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