| Pharmaceutical Information |
| Drug Name |
Risdiplam |
| Drug ID |
BADD_D02592 |
| 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 use in patients 2 months of age or older in 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. |
| Indications and Usage |
Risdiplam is indicated for the treatment of spinal muscular atrophy (SMA) in patients 2 months of age and older.[L15336] |
| Marketing Status |
approved; investigational |
| ATC Code |
M09AX10 |
| DrugBank ID |
DB15305
|
| KEGG ID |
D11406
|
| MeSH ID |
C000629884
|
| PubChem ID |
118513932
|
| TTD Drug ID |
D0N5MH
|
| NDC Product Code |
50242-175 |
| UNII |
76RS4S2ET1
|
| Synonyms |
Risdiplam | 7-(4,7-Diazaspiro(2.5)oct-7-yl)-2-(2,8-dimethylimidazo(1,2-b)pyridazin-6-yl)-4H-pyrido(1,2-a)pyrimidin-4-one |
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| Chemical Information |
| Molecular Formula |
C22H23N7O |
| CAS Registry Number |
1825352-65-5 |
| SMILES |
CC1=CC(=NN2C1=NC(=C2)C)C3=CC(=O)N4C=C(C=CC4=N3)N5CCNC6(C5)CC6 |
| Chemical Structure |
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| ADRs Induced by Drug |
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