STAT Plus: Biogen’s blockbuster rare disease drug could be in trouble

The success of Biogen’s spinal muscular atrophy treatment Spinraza has been among the few bright spots in a trying few years for the company. But new data from a competing medicine suggests diminishing returns in the future, a problem that could be exacerbated by the coronavirus pandemic.

On Tuesday, partners Roche and PTC Therapeutics disclosed that their SMA treatment, risdiplam, led to a 93% one-year survival rate among infants with the most severe form of the disease, which is almost universally fatal by age 2. In a similar study, Spinraza kept about 84% of severe SMA patients alive after a year.

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