2D 3D
	ID	DDInter1603
	Drug Type	small molecule
	Molecular Formula	C22H23N7O
	Molecular Weight	401.474
	CAS Number	1825352-65-5
	Description	Risdiplam is an orally bioavailable mRNA splicing modifier used for the treatment of spinal muscular atrophy (SMA).[L12615] It increases systemic SMN protein concentrations by improving the efficiency of _SMN2_ gene transcription. This mechanism of action is similar to its predecessor [nusinersen], the biggest difference being their route of administration: nusinersen requires intrathecal administration, as does the one-time gene therapy [onasemnogene abeparvovec], whereas risdiplam offers the ease of oral bioavailability.[L15351,A216871] Risdiplam was approved by the FDA in August 2020 for the treatment of spinal muscular atrophy (SMA).[L15331,L15336] Set to be substantially cheaper than other available SMA therapies,[L15351] risdiplam appears to provide a novel and relatively accessible treatment option for patients with SMA regardless of severity or type.
	ATC Classification	M09AX10
IUPAC Name	7-{4,7-diazaspiro[2.5]octan-7-yl}-2-{2,8-dimethylimidazo[1,2-b]pyridazin-6-yl}-4H-pyrido[1,2-a]pyrimidin-4-one
InChI	ASKZRYGFUPSJPN-UHFFFAOYSA-N
Canonical SMILES	CC1=CN2N=C(C=C(C)C2=N1)C1=CC(=O)N2C=C(C=CC2=N1)N1CCNC2(CC2)C1
Useful Links	DrugBank ChemSpider Wikipedia ChEMBL