Gene therapy company bluebird bio, Inc. (NASDAQ:BLUE) fell ~14% in the morning hours Thursday, ending week-long gains a day after the FDA approved its β-thalassemia drug Zynteglo making the treatment the priciest to date.
The FDA decision followed a June advisory committee meeting in which the agency’s independent experts voted 13-0 to recommend the drug’s approval.
Zynteglo, indicated for beta-thalassemia patients who require regular blood transfusions, is priced at $2.8M for a one-time therapy, making it the most expensive treatment in history.
Despite the cost, some analysts don’t expect the drug to become a significant revenue driver for the biotech as they point to the limited patient population and argue that many patients are unlikely to undergo this treatment.
BLUE has missed Street’s earnings forecasts for seven consecutive quarters, and its shares remain ~52% below the year-ago level, as illustrated in this graph.