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.
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