Ataluren

爱塔乐伦

Report date: 2026/03/31 20:00:21
NMEFirst In Class

Base Information

Drug NameAtaluren
Drug Alias Name阿他卢仑ataluren阿塔鲁伦atalurenoatalurenum +[7] 
ClassificationSmall molecules > Heterocyclic drugs
Mechanisms of ActionDystroglycan stimulants
TargetsDystroglycan
Therapy Area/IndicationsMethylmalonic acidaemiaBecker muscular dystrophyGenetic Diseases, X-Linked +[18] 
Global Approved IndicationsN/A
Approved Indications in ChinaN/A
Original Approved Country/TerritoryN/A
Original Approved DateN/A
Country/TerritoryCzechiaBelgiumBulgaria +[32] 
CombinationsNo
Global Highest StatusNDA/BLA
Highest Status in ChinaNDA/BLA
Originator CompaniesPtc Therapeutics Inc
China Registration ClassN/A

Develop Information

Active CompaniesPtc Therapeutics IncPtc Therapeutics International LtdFrosst Iberica Sa +[5] 
Inactive CompaniesGenzyme Corp
Active IndicationsGenetic Diseases, X-LinkedGenetic Diseases, InbornNeuromuscular Diseases +[9] 
Inactive IndicationsMethylmalonic acidaemiaBecker muscular dystrophyHemophilia B +[9] 

Other Information

ATC CodeM09AX03
Related LinksWikipediaDrugBankKEGG