35. Adakveo


Active ingredient: crizanlizumab
Disease: sickle cell disease
Peak sales estimate: over $1 billion
Approved: Nov. 15, 2019
Company: Novartis

The scoop: Early last year, Novartis unveiled a diverse slate of 25 potential drugs that it felt could each attain blockbuster status by 2021. It's moved closer to checking one of those boxes with Adakveo’s fast approval in sickle cell disease, as the first targeted therapy for vaso-occlusive crises: the painful clumps of sickled red blood cells that can block the flow of oxygen to organs, causing serious damage. The Swiss drugmaker first acquired the antibody in 2016 through its $665 million buyout of Selexys. It’s designed to reduce occlusions by inhibiting a protein that lines the surfaces of blood vessels and activated platelets, which also contributes to cell-cell adhesion. A phase 2 study showed it could cut annual rates by 45% and hospitalizations by 42% compared to placebo, with about 36% of participants having no crises within a 52-week period. Approved for patients 16 and older, Adakveo has a wholesale acquisition cost of $2,357 per vial. Based on weight, most patients would take three or four vials a month. — Conor Hale

35. Adakveo

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