Chemical formula: C₂₂H₂₃N₇O Molecular mass: 401.474 g/mol
Risdiplam is a survival of motor neuron 2 (SMN2) pre-mRNA splicing modifier designed to treat SMA caused by mutations of the SMN1 gene in chromosome 5q that lead to SMN protein deficiency. Functional SMN protein deficiency is directly linked to the SMA pathophysiology which includes progressive loss of motor neurons and muscle weakness. Risdiplam treats SMA by increasing and sustaining functional SMN protein levels.
This medicinal substance has been classified in the anatomical therapeutic chemical (ATC) classification according to its main therapeutic use as follows:
| ATC code | Group title | Classification |
|---|---|---|
| M09AX10 | M Musculo-skeletal system → M09 Other drugs for disorders of the musculo-skeletal system → M09A Other drugs for disorders of the musculo-skeletal system → M09AX Other drugs for disorders of the musculo-skeletal system | |
Competent medicine agencies globally have authorized commercialization of this active ingredient according to these medication package inserts (MPIs):
| Title | Information Source | Document Type | |
|---|---|---|---|
| EVRYSDI Powder for oral solution | European Medicines Agency (EU) | MPI, EU: SmPC | |
| EVRYSDI Oral solution | FDA, National Drug Code (US) | MPI, US: SPL/PLR |
Risdiplam is an active ingredient of these brands:
United States (US)Brazil (BR)Croatia (HR)Ecuador (EC)Estonia (EE)Finland (FI)Hong Kong (HK)Ireland (IE)Israel (IL)Japan (JP)Lithuania (LT)New Zealand (NZ)Poland (PL)Romania (RO)Spain (ES)United Kingdom (UK)Note the following: The list of brand names is continuously updated, and thus does not include the total of products circulating worldwide. |
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