56. Ultomiris


Active ingredient: ravulizumab
Disease: paroxysmal nocturnal hemoglobinuria
Peak sales estimate: $5 billion by 2022 (for Soliris and Ultomiris combined)
Approved: Dec. 21
Company: Alexion

The scoop: Alexion is out with a new-and-improved version of lead drug Soliris, and it’s not only more effective and more convenient—it’s cheaper, too. The company priced Ultomiris at a 10% discount to Soliris, whose maintenance doses run about $508,000 in rare disease paroxysmal nocturnal hemoglobinuria. And if Ultomiris can grab some of Soliris’ other indications, that discount could grow to 30%, Leerink Partners analyst Geoffrey Porges says. Meanwhile, Alexion will be working to switch patients over to the newer drug, which is dosed every eight weeks; its predecessor requires maintenance doses every two weeks. — Carly Helfand

56. Ultomiris

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