Active ingredient description

A nonsense mutation in DNA results in a premature stop codon within an mRNA. This premature stop codon in the mRNA causes disease by terminating translation before a full-length protein is generated. Ataluren enables ribosomal readthrough of mRNA containing such a premature stop codon, resulting in production of a full-length protein.

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
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 M09AX03

Product monographs

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

Summary of Product Characteristics (SPC)


Ataluren is the active ingredient of these drugs:


Austria Brazil Estonia Finland France

Structural formula

Graphic representation of the active ingredient's molecular structure

Chemical formula: C₁₅H₉FN₂O₃
Molecular mass: 284.242 g/mol

External identifiers

CAS Substance: 775304-57-9
DrugBank Drug: DB05016
KEGG Drug: D09323
PubChem Compound: 11219835
SNOMED-CT Concept: 718843009
Ataluren (substance)
UNII Identifier: K16AME9I3V