10. Spinraza

Analysts are looking for quick blockbuster sales from Spinraza, but the drug's $750,000 price tag for the first year of therapy brought Biogen and Ionis some unwelcome attention.

Generic name: nusinersen
Company: Biogen and Ionis
Disease: spinal muscular atrophy
2022 sales estimate: $1.3 billion

Biogen and Ionis nabbed an earlier-than-expected approval for Spinraza last month and quickly tagged their first-ever med for spinal muscular atrophy with a $750,000 price for an initial year's therapy and $375,000 per year after that.

The per-year sticker isn't outside the range typical for rare disease treatments, which Spinraza is, but amid the intensified scrutiny on drug prices—and alongside that $750,000 tag for the first year—the announcement attracted plenty of attention. And that fact puts Spinraza off to an unaccustomed start.

The drug's price prompted some consternation on Twitter, and shares in Biogen and Ionis both dropped on the news. Some tweets urged action from lawmakers, including now-President Donald Trump. Analysts wondered whether the public response to Spinraza's price will embolden payers to press Biogen for discounts. 

Leerink Partners analyst Geoffrey Porges took that worry a step further, suggesting that Spinraza's price could draw unwanted attention to the cost of other rare disease meds. “The sticker-shock presented in the media could turn Spinraza into the Sovaldi of rare disease drugs,” Porges wrote to investors after the announcement. It could be “the straw that breaks the camel’s back in terms of the U.S. market’s tolerance for rare disease drug pricing.”

Despite the debate, however, analysts expect Spinraza to be a success for Biogen and Ionis. Porges himself raised his sales expectations for the drug to $2 billion by 2020, and he said he expects 1,700 patients to be treated this year. And Piper Jaffray analyst Joshua Schimmer said, "We expect Spinraza's launch to exceed expectations."

10. Spinraza