efzimfotase alfa efzimfotase alfa PHASE3
Drug Profile
ModalityEnzyme
RouteSC
Therapy AreaRare Disease
Peak Sales Est$800M
Formulations[]
Companies
AZN (ORIGINATOR)100%
Mechanism: Tissue-nonspecific ALP
Expert: Bone-targeted enzyme replacement therapy using Fc-deca-aspartate fusion of tissue-nonspecific alkaline phosphatase for hypophosphatasia.
Everyday: Replaces a missing enzyme that helps bones and teeth develop properly.
Targets: ["TNSALP"]
Programs (1)
IndicationStageKey StudyRegional Status
HPPPHASE3LIFT[]
Upcoming Catalysts (1)
Efzimfotase Alfa - HPP - Ph3 - Topline H1 2026
Notes
Enzyme replacement for hypophosphatasia.
Data from Supabase · Updated 2026-03-24