A gene therapy will be offered for sale in Europe with a money-back guarantee, according to GlaxoSmithKline, the company commercializing it.
The treatment, called Strimvelis, is the first outright cure for a rare disorder to emerge from gene therapy, and its price tag of 594,000 euros ($665,000)…makes it one of the most expensive one-time treatments ever sold by a drug firm.
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By some measures, Strimvelis’s price counts as a bargain. The cost of a bone marrow transplant from another person…can reach $1 million…The expense of these drugs and the care needed for a sick child quickly add up to millions.
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“I was expecting a higher price,” says Christian Hill…“My initial gut feeling was ‘Hmmm, that’s really surprising.’”
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But the big question isn’t whether gene therapy costs too much—it’s whether companies can make any money at it, especially treating ultra-rare diseases. Only about a dozen children are born with ADA-SCID each year in Europe. Treating all of them would generate about $8 million in revenue—barely a blip for GSK, which sells $30 billion worth of drugs a year.
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