Risdiplam

Risdiplam
Clinical data
Trade namesEvrysdi
Other namesRG7916; RO7034067
AHFS/Drugs.comMonograph
License data
Pregnancy
category
Routes of
administration		By mouth
ATC code
Legal status
Legal status
Identifiers
  • 7-(4,7-diazaspiro[2.5]octan-7-yl)-2-(2,8-dimethylimidazo[1,2-b]pyridazin-6-yl)pyrido[1,2-a]pyrimidin-4-one
CAS Number
PubChem CID
DrugBank
ChemSpider
UNII
KEGG
ChEMBL
CompTox Dashboard (EPA)
ECHA InfoCard100.278.103 Edit this at Wikidata
Chemical and physical data
FormulaC22H23N7O
Molar mass401.474 g·mol−1
3D model (JSmol)
  • CC1=CC(=NN2C1=NC(=C2)C)C3=CC(=O)N4C=C(C=CC4=N3)N5CCNC6(C5)CC6
  • InChI=1S/C22H23N7O/c1-14-9-18(26-29-11-15(2)24-21(14)29)17-10-20(30)28-12-16(3-4-19(28)25-17)27-8-7-23-22(13-27)5-6-22/h3-4,9-12,23H,5-8,13H2,1-2H3
  • Key:ASKZRYGFUPSJPN-UHFFFAOYSA-N

Risdiplam, sold under the brand name Evrysdi, is a medication used to treat spinal muscular atrophy (SMA)[6][9] and the first oral medication approved to treat this disease.[6][9]

Risdiplam is a survival of motor neuron 2-directed RNA splicing modifier.[6][5][10]

In clinical trials, the most common adverse events included fever, diarrhea, rash, ulcers of the mouth area, joint pain (arthralgia) and urinary tract infections.[6][5] Additional adverse events observed in the infantile-onset population included upper respiratory tract infection, pneumonia, constipation and vomiting.[6][5]

Risdiplam was approved by the US Food and Drug Administration (FDA) in August 2020, for the treatment of adults and children two months of age or older.[6][11] Developed by Roche in Basel, Switzerland,[12] in association with PTC Therapeutics and the SMA Foundation,[9][11] it is marketed in the US by Genentech,[6] a subsidiary of Roche.[11]

  1. ^ a b "Evrysdi". Therapeutic Goods Administration (TGA). 11 June 2021. Retrieved 6 September 2021.
  2. ^ a b "AusPAR: Risdiplam". Therapeutic Goods Administration (TGA). 13 September 2021. Retrieved 13 September 2021.
  3. ^ "Summary Basis of Decision (SBD) for Evrysdi". Health Canada. 23 October 2014. Retrieved 29 May 2022.
  4. ^ "Health product highlights 2021: Annexes of products approved in 2021". Health Canada. 3 August 2022. Retrieved 25 March 2024.
  5. ^ a b c d Cite error: The named reference Evrysdi label was invoked but never defined (see the help page).
  6. ^ a b c d e f g h O'Keefe L (7 August 2020). "FDA Approves Oral Treatment for Spinal Muscular Atrophy" (Press release). Silver Spring, Maryland, United States of America: United States Food and Drug Administration (FDA). FDA Newsroom Department. Archived from the original on 11 August 2020. Retrieved 7 August 2020. Public Domain This article incorporates text from this source, which is in the public domain.
  7. ^ "Evrysdi EPAR". European Medicines Agency. 24 February 2021. Retrieved 4 March 2023.
  8. ^ "Evrysdi Product information". Union Register of medicinal products. Retrieved 3 March 2023.
  9. ^ a b c Food and Drugs Administration (FDA) (7 August 2020). "Evrysdi (Risdiplam) for Spinal Muscular Atrophy". SMA News Today. Pensacola, Florida, United States: BioNews Services (BioNews Services, LLC.). Archived from the original on 27 January 2021. Retrieved 9 June 2021.
  10. ^ Cite error: The named reference Zhao was invoked but never defined (see the help page).
  11. ^ a b c Cite error: The named reference Genentech PR was invoked but never defined (see the help page).
  12. ^ Cite error: The named reference Ratni Ebeling Baird Bendels pp. 6501–6517 was invoked but never defined (see the help page).