Risdiplam
Active Pharmaceutical Ingredients 2025-03-03
English Synonyms RG-7916 RO-7034067 2-(2,8-Dimethylimidazo[1,2-b]pyridazin-6-yl)-7-(4,7-diazaspiro[2.5]octan-7-yl)-4H-pyrido[1,2-a]pyrimidin-4-one
Catalog ID101710 CAS NO.1825352-65-5
Purity 95%
MDL NumberMFCD31657372
EINEC Molecular Formula C22H23N7O
Molecular Weight 401.46
| Category | Content |
|---|---|
| Generic Name | Risdiplam |
| Brand Name | Evrysdi |
| Dosage Form | Oral powder for reconstitution (0.75 mg/mL) |
| Drug Class | mRNA splicing modifier |
| Mechanism of Action | Risdiplam is an mRNA splicing modifier that targets the SMN2 gene to increase the production of functional SMN protein, which is deficient in patients with spinal muscular atrophy (SMA). |
| Indications | Treatment of spinal muscular atrophy (SMA) in patients aged 2 months and older. |
| Side Effects | Common side effects include fever, lung infection, vomiting, diarrhea, constipation, rash, and cold symptoms. |
| Storage | Store in a refrigerator (2°C to 8°C). Do not freeze. Can be kept at room temperature (below 40°C) for up to 5 days. |
| Administration | Oral or via feeding tube, once daily after a meal. |
| Developer | Genentech (a member of the Roche Group) |
| Approval Date | FDA approval in August 2020. |
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