Gene therapy has already changed medicine. Now it might get some competition

In the U.S., there are two approved gene therapies. Each set a record for the cost of a single medicine, and each manufacturer had a compelling defense for that cost: In one case, the alternative is blindness; in the other, it’s certain death.

But what about when the alternative is simply a different gene therapy? That could soon be the reality for patients with hemophilia A, as three one-time treatments are moving toward the market.

At a medical conference over the weekend, BioMarin said it’s on track to file for FDA approval by the end of this year. Spark Therapeutics is about a year behind with a gene therapy of its own. And Sangamo Therapeutics, partnered with Pfizer, presented promising early data suggesting it could compete with the other two in about two years.

If each company succeeds, hemophilia A will become the first competitive market for gene therapy, which — if standard economic principles have any application in the drug industry — should have an effect on pricing.