Risdiplam

Risdiplam
Clinical data
Trade names	Evrysdi
Other names	RG7916; RO7034067
AHFS/Drugs.com	Monograph
License data	US DailyMed: Risdiplam;
Pregnancy; category	AU: D;
Routes of; administration	By mouth
ATC code	M09AX10 (WHO) ;
Legal status
Legal status	AU: S4 (Prescription only); CA: ℞-only; US: ℞-only; EU: Rx-only;
Identifiers
	IUPAC name 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	1825352-65-5;
PubChem CID	118513932;
DrugBank	DB15305;
ChemSpider	67886354;
UNII	76RS4S2ET1;
KEGG	D11406;
ChEMBL	ChEMBL4297528;
CompTox Dashboard (EPA)	DTXSID701109185 ;
ECHA InfoCard	100.278.103
Chemical and physical data
Formula	C22H23N7O
Molar mass	401.474 g·mol−1
3D model (JSmol)	Interactive image;
	SMILES CC1=CC(=NN2C1=NC(=C2)C)C3=CC(=O)N4C=C(C=CC4=N3)N5CCNC6(C5)CC6;
	InChI 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) and is the first oral medication approved to treat this disease by the US Food and Drug Administration (FDA).

Risdiplam is a survival of motor neuron 2-directed RNA splicing modifier.

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

Risdiplam was approved for medical use in the United States in August 2020. Developed by Roche in Basel, Switzerland, in association with PTC Therapeutics and the SMA Foundation, it is marketed in the US by Genentech, a subsidiary of Roche. In February 2025, the FDA approved a new tablet formulation of risdiplam.