Sepofarsen

N/A

Report date: 2025/06/26 16:54:53
NME

Base Information

Drug NameSepofarsen
Drug Alias NameQR-110EW-9I3PJC3R
ClassificationSmall molecules > Nucleosides and nucleotide derivtives > Oligonucleotide > Oligonucleotide antisense
Mechanisms of ActionGene expression stimulantsCentrosomal protein of 290 kDa modulators
TargetsCentrosomal protein of 290 kDa
Therapy Area/IndicationsRetinal DystrophiesRetinal DiseasesRetinal Degeneration +[11] 
Global Approved IndicationsN/A
Approved Indications in ChinaN/A
Original Approved Country/TerritoryN/A
Original Approved DateN/A
Country/TerritoryBelgiumCanadaUnited States +[9] 
CombinationsNo
Global Highest StatusPhase 3 Clinical
Highest Status in ChinaN/A
Originator CompaniesProqr Therapeutics
China Registration ClassN/A

Develop Information

Active CompaniesSepul BioProqr TherapeuticsLaboratoires Thea SAS
Inactive CompaniesN/A
Active IndicationsRetinal DiseasesLeber Congenital AmaurosisEye Diseases, Hereditary +[8] 
Inactive IndicationsRetinal DystrophiesRetinal DegenerationEye Diseases

Other Information

ATC CodeN/A
Related LinksN/A