.Pharmacolibrary.Drugs.M_MusculoSkeletalSystem.M09A_OtherDrugsForDisordersOfTheMusculoSkeletalSystem.M09AX10_Risdiplam.Risdiplam .Pharmacolibrary.Drugs.M_MusculoSkeletalSystem.M09A_OtherDrugsForDisordersOfTheMusculoSkeletalSystem.M09AX10_Risdiplam.Risdiplam| name: | Risdiplam |
| ATC code: | M09AX10 | route: | oral |
| n-compartments | 2 |
Risdiplam is an orally administered small molecule used for the treatment of spinal muscular atrophy (SMA). It acts as a survival motor neuron 2 (SMN2) splicing modifier, increasing production of functional SMN protein. Risdiplam is currently approved by the FDA and EMA for use in both pediatric and adult patients with SMA.
Pharmacokinetic parameters reported in healthy adult volunteers and SMA patients, after oral administration.
Cleary, Y, et al., & Gertz, M (2023). Estimation of FMO3 Ontogeny by Mechanistic Population Pharmacokinetic Modelling of Risdiplam and Its Impact on Drug-Drug Interactions in Children. Clinical pharmacokinetics 62(6) 891–904. DOI:10.1007/s40262-023-01241-7 PUBMED:https://pubmed.ncbi.nlm.nih.gov/37148485