Lessons abound in the story about Glybera, a gene therapy developed by the Dutch biotech company UniQure to treat a very rare disease, but one stands out: Good luck getting insurers to cover a treatment that costs $1 million for a one-time dose and that helps only a miniscule number of patients. In this case that number is one. Glybera, the first gene therapy put on the market in the Western world, treats familial lipoprotein lipase deficiency, a one-in-a-million rare disease, and has been used on one patient since its approval in 2012 by the European Medicines Agency. UniQure, faced with these market realities, has decided to let the approval for Glybera lapse, according to STAT.
Perhaps the biggest surprise is that proponents of gene therapy have not allowed Glybera’s fate to dampen their enthusiasm about other such therapies in the works. Some of those therapies are being developed by Spark Therapeutics, Bluebird Bio, and BioMarin Pharmaceuticals.
In perhaps one of the bigger understatements of the week, Stuart Orkin, MD, a hematologist at Boston Children’s Hospital, tells STAT: “This first therapy is not a great example to start with. Therapies that work well, I believe, will be supported in the marketplace, as they will benefit patients and save medical costs over the long haul.”
So what else is in the works? Spark says its gene therapy can improve vision in patients with rare eye diseases, and it looks as if that therapy will be approved by the FDA possibly this year. Meanwhile, according to STAT, “BioMarin is at work on a gene therapy for hemophilia with plans to begin this year a trial that could support FDA approval. And Bluebird is working through a late-stage trial of its treatment for a rare blood disease, the results of which will go straight to regulators if they’re positive.”