Risdiplam

Molecular mass: 401.474 g/mol 

Active ingredient description

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.

Medicine classification

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
Discover more medicines within M09AX10

Product monographs

Competent medicine agencies globally have authorized commercialization of this active ingredient according to these medication package inserts (MPIs):

Title Information Source Document Type  
EVRYSDI Oral solution FDA, National Drug Code (US) MPI, US: SPL/PLR
EVRYSDI Powder for oral solution European Medicines Agency (EU) MPI, EU: SmPC

Structural formula

Graphic representation of the active ingredient's molecular structure

External identifiers

CAS Substance: 1825352-65-5
DrugBank Drug: DB15305
KEGG Drug: D11406
RxNorm Ingredient: 2390935
UNII Identifier: 76RS4S2ET1
RISDIPLAM

Medicines

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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