Cancer is probably the most sought-after indication in the biopharma world, but it’s missing from 2019’s class of top drug launches. Instead, treatments in immunology and rare genetic diseases have grabbed the limelight.
We considered two EvaluatePharma projections in January and February to rank this year’s top 10 drug launches by 2024 sales. At the top of the list is Alexion’s Soliris follow-up, Ultomiris, which nabbed an FDA nod at the end of 2018, two months ahead of its scheduled decision date.
As part of CEO Ludwig Hantson’s plan for Alexion to pivot away from ultrarare diseases and reach more patients, the biotech has priced Ultomiris at a 10% discount to Soliris and is aiming to convert70% of Soliris patients to the improved therapy. Paroxysmal nocturnal hemoglobinuria (PNH), the current condition Ultomiris is approved in, is a rare disease that involves the immune system.
Notably, two gene therapies are included here: Novartis’ spinal muscular atrophy treatment Zolgensma—which could challenge Biogen’s SMA treatment Spinraza—and Bluebird Bio’s Zynteglo for beta-thalassemia. Gene therapies have attracted much interest and controversy alike. As one-time treatments, they come with big efficacy promises, but pricing of these two drugs has been closely watched, and both companies are offering up novel models such as installments paid over several years.
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