Multimillion-dollar gene therapies are 'in the right ballpark': drugmaker exec

BlueBird Bio's two drugs for rare diseases that each cost about $3 million — the most expensive drugs on the market — are fairly priced, the chief operating officer at a rival drugmaker told Bloomberg in an Oct. 5 report. 

Bluebird, a Cambridge, Mass.-based biotech company, prices its rare autosomal recessive disorder treatment for $2.8 million and another gene therapy drug for $3 million. Both medications are intended for one-time use, but because the pool of potential customers is small, the price tag may not be enough to keep the drugmaker in business. 

"Do I think those sorts of prices are in the right ballpark for a one-time curative therapy for something like this?" Stuart Arbuckle, chief operating officer of Vertex Pharmaceuticals, which is developing a sickle-cell disease drug, told Bloomberg. "Yeah, I think those are in the right ballpark."

Vertex told Bloomberg that Mr. Arbuckle's comments should not be read as a hint toward the price of its genetic therapy drug, which has not been released. But the Institute for Clinical and Economic Review seems to agree with this "ballpark" figure.

In July, the ICER said Zynteglo, Bluebird's $2.8 million treatment for beta thalassemia, which affects 1 in 100,000 people, should cost $2.1 million "given the high costs of standard care."

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