.Pharmacolibrary.Drugs.M_MusculoSkeletalSystem.M09A_OtherDrugsForDisordersOfTheMusculoSkeletalSystem.M09AX07_Nusinersen.Nusinersen

Information

Nusinersen is an antisense oligonucleotide drug approved for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients. It works by modifying the splicing of SMN2 pre-mRNA to increase production of full-length survival motor neuron (SMN) protein. Nusinersen is currently approved and administered globally for SMA.

Pharmacokinetics

Pharmacokinetic parameters observed in pediatric and adult patients with spinal muscular atrophy following intrathecal administration.

References

1. Luu, KT, et al., & Wang, Y (2017). Population Pharmacokinetics of Nusinersen in the Cerebral Spinal Fluid and Plasma of Pediatric Patients With Spinal Muscular Atrophy Following Intrathecal Administrations. Journal of clinical pharmacology 57(8) 1031–1041. DOI:10.1002/jcph.884 PUBMED:https://pubmed.ncbi.nlm.nih.gov/28369979

2. MacCannell, D, et al., & Finkel, RS (2021). Population pharmacokinetics-based recommendations for a single delayed or missed dose of nusinersen. Neuromuscular disorders : NMD 31(4) 310–318. DOI:10.1016/j.nmd.2021.02.014 PUBMED:https://pubmed.ncbi.nlm.nih.gov/33781694

3. MacCannell, D, et al., & Muntoni, F (2022). Restoration of Nusinersen Levels Following Treatment Interruption in People With Spinal Muscular Atrophy: Simulations Based on a Population Pharmacokinetic Model. CNS drugs 36(2) 181–190. DOI:10.1007/s40263-022-00899-0 PUBMED:https://pubmed.ncbi.nlm.nih.gov/35080757

Revisions

• 06/2025 initial generated model