11. Zolgensma


Active ingredient: onasemnogene abeparvovec
Disease: spinal muscular atrophy
Peak sales estimate: $1.9 billion
Approved: May 24, 2019
Company: Novartis

The scoop: Novartis' gene therapy Zolgensma was one of the top headline-producers of the year after its monumental FDA nod, its record price, a data manipulation scandal and more. The spinal muscular atrophy gene therapy scored FDA approval in late May and with the approval, Novartis set the price at $2.125 million. A one-time medicine intended to cure patients, the drug scored an endorsement from cost watchdogs at ICER despite the record price. Also this year, Novartis suffered a scandal surrounding the drug after it became public that researchers with AveXis—a gene therapy company that Novartis acquired—manipulated data in preclinical development. Novartis and the FDA concluded that the manipulation didn't affect safety or efficacy. Sales came in strong during the drug's first full quarter on the market as Novartis reported $160 million in third-quarter Zolgensma revenues, beating analyst expectations by 60%. — Eric Sagonowsky

11. Zolgensma

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