INZ-701
Withdrawn or never marketedHeight measured in peopleBy injectionProtein or enzymeAlso BMN 401
The missing ENPP1 enzyme manufactured as an injected drug, for the inherited disease where losing that enzyme calcifies the arteries in infancy and causes rickets later. It was never approved and has been abandoned.
Recombinant human ENPP1-Fc protein: the extracellular domains of human ENPP1 coupled to an Fc fragment from an immunoglobulin gamma-1 antibody, injected under the skin as an enzyme replacement for ENPP1 deficiency — generalised arterial calcification of infancy and autosomal recessive hypophosphataemic rickets type 2. It came out of Inozyme Pharma and was carried forward by BioMarin as BMN 401. No regulator approved it anywhere, both phase 3 studies are recorded as terminated, and the sponsor has discontinued development.
Risks. It was never approved, so no regulator set out its risks and there are no official warnings. In the first study in people, 13 adults with the disease, nobody died and three had a serious problem: a fainting episode, a broken kneecap and an operation called off. Ten of the thirteen made antibodies against the drug itself.
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