Mr. Ibrahem/Risdiplam
Clinical data
Trade namesEvrysdi
Other namesRG7916; RO7034067
AHFS/Drugs.comMonograph
License data
Routes of
administration
By mouth
Drug classSMN2-directed RNA splicing modifier[1]
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
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).[1] This includes type 1, type 2, and type 3 disease.[3] It is used in those who are at least two months old.[1] It is taken by mouth.[1]

Common side effects included fever, diarrhea, rash, pneumonia, and vomiting.[1] Use in pregnancy may harm the baby.[4] It is a survival of motor neuron 2-directed RNA splicing modifier.[1]

Risdiplam was approved for medical use in the United States in 2020 and Europe in 2021.[5][3] In the United States 60 mg costs about 11,700 USD as of 2021.[6] In the United Kingdom this amount costs the NHS about £7900.[7]

References

edit
  1. ^ a b c d e f g h i j Food and Drugs Administration (FDA) (18 August 2020). "Evrysdi- risdiplam powder, for solution". DailyMed. Bethesda, Maryland, United States: MedLine by the National Library of Medicine (NLM) of the United States National Institutes of Health (NIH). Archived from the original on 29 August 2021. Retrieved 24 September 2020.
  2. ^ O'Keefe, Lindsey (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.   This article incorporates text from this source, which is in the public domain.
  3. ^ a b "Evrysdi". Archived from the original on 6 October 2021. Retrieved 18 October 2021.
  4. ^ "Risdiplam (Evrysdi) Use During Pregnancy". Drugs.com. Archived from the original on 30 October 2020. Retrieved 18 October 2021.
  5. ^ "Risdiplam Monograph for Professionals". Drugs.com. Archived from the original on 20 October 2021. Retrieved 18 October 2021.
  6. ^ "Evrysdi Prices, Coupons & Patient Assistance Programs". Drugs.com. Retrieved 18 October 2021.
  7. ^ "Risdiplam". SPS - Specialist Pharmacy Service. 6 February 2019. Archived from the original on 18 October 2021. Retrieved 18 October 2021.