miércoles, 25 de marzo de 2020

Novartis might win the race in SMA

The Readout
Damian Garde & Meghana Keshavan

Novartis might win the race in SMA

For patients with spinal muscular atrophy, there’s a gene therapy for infants with severe disease, an injectable medicine for older people, and a not-yet-approved oral treatment with promise for milder cases. But that entire dynamic might shift thanks to new data from Novartis.

The company’s gene therapy, Zolgensma, met its goals in a small study involving patients with Type 2 SMA, less severe than the indication for which it’s already approved. Novartis’s data compare favorably with the benefits of Biogen’s injectable Spinraza and Roche’s oral risdiplam, according to RBC analyst Brian Abrahams.

If Novartis can consistently outperform its rivals in more SMA sub-populations, the one-time therapy could become the best-in-class treatment, according to Abrahams, altering a market long thought to be competitive.

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