Biogen took a lot of analysts by surprise recently when the big biotech announced that its breakthrough drug for spinal muscular atrophy would be priced at $750,000 for the first year, and $375,000 for each subsequent year. But investigators just spelled out the data from their pivotal study, demonstrating a clear reduction in the risk of death.
While the study was stopped early due to its success, 68% of the untreated infants in the study either died or required permanent ventilation compared to 39% of the drug arm. That represented a 47% drop in the risk of death or need for permanent ventilation.
The data were presented at the British Paediatric Neurology Association annual conference in Cambridge, UK.
This drug, and the steep price tied to it, is firing up the debate over pricing new drugs at a time the industry has begun to seriously grapple with the controversial issue for the first time. Landmark new drugs are generating sticker shock over the wholesale price, but also demonstrate the potential for major medical advances for patients.
That’s one of several stripes of controversies that will be aired in Congress over the course of 2017, after president-elect Donald Trump told the world this week that pharma companies have been “getting away with murder” on prices. Lawmakers have been struggling to come up with new rules to prevent price gouging on old therapies while the backlash against annual or semi-annual increase in portfolio drugs led a growing lineup of biopharmas to pledge price discipline.
“We are very encouraged that individuals with SMA have already started treatment with Spinraza this week in the U.S., and we continue to work closely with regulatory agencies to bring this therapy to patients around the world as quickly as possible,” said Wildon Farwell, MD, MPH., senior medical director, clinical development, at Biogen.
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