MT1621
doxecitine/doxribtimine
APPROVED
Drug Profile
ModalitySmall molecule
RouteOral
Therapy AreaRare Disease
Peak Sales Est$300M
Formulations[]
Mechanism: Nucleoside therapy
Expert: Provides deoxycytidine and deoxythymidine to bypass enzyme deficiency
Everyday: Provides deoxycytidine and deoxythymidine to bypass enzyme deficiency
Targets: []
Programs (1)
Notes
Deoxynucleoside substrate enhancement therapy for thymidine kinase 2 (TK2) deficiency, a mitochondrial DNA depletion syndrome. First potential treatment for this ultra-rare, fatal myopathy affecting children.
Data from Supabase · Updated 2026-03-24