LONDON — The United Kingdom’s thrifty health authority has cleared its most expensive medicine ever — a $700,000 GlaxoSmithKline PLC treatment for an illness known as “bubble boy disease.’’ But the overall cost to the country’s medical system won’t be as eye-popping as the price suggests.
Glaxo, Britain’s biggest drug maker, said by e-mail it has treated four patients with Strimvelis since it was approved by the European Union in May 2016.
The one-time procedure and potential cure, targeting an ultra-rare disease that leaves newborn babies unable to fight off everyday infections, is priced at 594,000 euros ($716,000).
Strimvelis, only the second gene therapy for an inherited disease to be licensed anywhere in the world, was recommended Wednesday by Britain’s National Institute for Health and Care Excellence, which determines whether new drugs are cost-effective.
While gene therapies are revolutionizing treatment for an array of diseases after overcoming decades of challenges, questions remain about their commercial prospects and pricing.
Spark Therapeutics Inc. last month won US approval of its gene therapy for an inherited form of blindness. Since the therapy is intended to be administered only once, Spark said it will charge $850,000 for both eyes.
The company says two more patients are due to be treated soon with Strimvelis, currently available only at a hospital in Milan. The patient numbers reflect the rare incidence of the disease, a spokesman for the London-based company said in an e-mail. The treatment is not available in the United States.
About three babies a year in England are born with the condition, called severe combined immunodeficiency syndrome due to adenosine deaminase deficiency, or ADA-SCID. It’s informally known as “bubble boy disease’’ after a 1976 film starring John Travolta about a patient living in a sterile environment.
While stem cell transplants, the current treatment, can restore the immune system, they may not succeed in all cases, and closely matched donors are hard to find or may not be available, according to the United Kingdom’s drug price monitor.
