Bluebird Bio sells two of the most expensive drugs on the market, both priced at about $3 million per patient, but they may not be enough to keep the business afloat, The Wall Street Journal reported Oct. 3.
Since August, the FDA has approved two of the Cambridge, Mass.-based biotech company's gene therapy drugs — Zynteglo, priced at $2.8 million, and Skysona, priced at $3 million — but they both treat rare diseases, which could hinder revenue.
Zynteglo is a treatment for beta thalassemia in patients who require regular red blood cell transfusions, but the autosomal recessive disorder only affects 1 in 100,000 people, according to the National Organization for Rare Disorders.
Skysona is on the FDA's accelerated approval track to treat boys 4 to 17 years old with early, active cerebral adrenoleukodystrophy. ALD is a genetic condition present among 1 in every 21,000 males born, according to Boston Children's Hospital, and CALD develops in 40 percent of that population.
Both drugs are one-time treatments, but because both conditions are rare, Bluebird may have trouble raking in the profits it needs. The biotech company plans to spend about $340 million this year, but at the end of its second quarter, it only had about $218 million in cash, according to the Journal.
Read the full report here.