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.

The risdiplam data is good news for patients, who will soon have three options for treatment, including a gene therapy from Novartis. The risk for Biogen, however, is that Spinraza might be the least popular.

STAT+ Exclusive Story

Already have an account? Log in

This article is exclusive to STAT+ subscribers

Unlock this article — plus daily coverage and analysis of the biotech sector — by subscribing to STAT+.

Already have an account? Log in

Individual plans

Group plans

Monthly

$39

Totals $468 per year

$39/month Get Started

Totals $468 per year

Starter

$30

for 3 months, then $399/year

$30 for 3 months Get Started

Then $399/year

Annual

$399

Save 15%

$399/year Get Started

Save 15%

11+ Users

Custom

Savings start at 25%!

Request A Quote Request A Quote

Savings start at 25%!

2-10 Users

$300

Annually per user

$300/year Get Started

$300 Annually per user

View All Plans

To read the rest of this story subscribe to STAT+.

Subscribe

Log In