Evrysdi

USDailyMed:Risdiplam AU:D M09AX10(WHO) AU:S4(Prescription only)CA:℞-onlyUS:℞-onlyEU:Rx-only 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 1825352-65-5 118513932 DB15305 67886354 76RS4S2ET1 D11406 ChEMBL4297528 DTXSID701109185 Interactive image 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,5-8,13H2,1-2H3Key:ASKZRYGFUPSJPN-UHFFFAOYSA-N Risdiplam, sold under the brand nameEvrysdi, is a medication used to treatspinal muscular atrophy(SMA)and is the firstoral medicationapproved to treat this disease by the USFood and Drug Administration(FDA). Risdiplam is asurvival of motor neuron 2-directedRNA splicingmodifier. 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 byRochein Basel, Switzerland,in association withPTC Therapeuticsand the SMA Foundation,it is marketed in the US byGenentech,a subsidiary ofRoche.In February 2025, the FDA approved a new tablet formulation of risdiplam.