The World’s Most Expensive Medicine Is a Bust
The first gene therapy approved in the Western world costs $1 million and has been used just once. The doctor who tried it says the price is “absolutely too high.”
by Antonio Regalado May 4, 2016
The most expensive drug in history is a money loser that’s not reaching patients. In fact, it’s only been paid for and used commercially once since being approved in 2012.
The medication in question is alipogene tiparvovec, better known as Glybera, a medicine widely heralded as the “first gene therapy” in the Western world and whose approval helped ignite an explosion of investment and excitement around treatments that correct DNA.
But when the Berlin physician Elisabeth Steinhagen-Thiessen wanted to give a patient Glybera last fall, it wasn't so easy. She says she had to prepare a submission as thick as “a thesis” for German regulators and then personally call the CEO of DAK, one of Germany’s large sickness funds, or insurers, to ask him to pay the $1 million price tag.
Last September, she gave 40 injections to the muscles of a 43-year-old woman with an ultra-rare disease called lipoprotein lipase deficiency. Such patients don’t process fat correctly. “You draw blood and you are astonished, there is no red blood, it's cream,” Steinhagen-Thiessen says. One symptom is debilitating abdominal pain. Her patient had been hospitalized more than 40 times.
A dose of Glybera contains trillions of viruses harboring correct copies of the lipoprotein lipase gene. And Steinhagen-Thiessen says the treatment, at Charite Hospital in Berlin, was a success. The woman hasn’t been back to the emergency room since the treatment and is now “living like you and me.”
But this single use of the drug just proves that Glybera is a flop. The problem is its staggering million-dollar price tag, too few patients, and questions about how effective it is.
The company that developed Glybera, UniQure, based in Amsterdam and Lexington, Mass., last fall dropped plans to get it approved in the U.S. and has turned over European sales to the Italian drug maker Chiesi Farmaceutici, which calls selling the drug "challenging."
“I think we learned a tremendous amount about what to do and what not to do, but commercially it has not been a success. It still drains a lot from the company,” says Dan Soland, UniQure's CEO. UniQure is now focused on developing other gene therapies, including one to treat hemophilia.
How Glybera turned into a money loser is a cautionary tale for gene therapy, a resurgent technology that has been drawing investor interest because of its promise to cure rare, inherited diseases with one-time repairs to a person’s DNA. A single dose of gene therapy can change the genetic instructions inside a person's cells in ways that last many years, or even a lifetime.
In addition to Glybera, there is at least one form of gene therapy approved in China to treat cancer by adding a gene to tumors, and late last year Amgen won U.S. approval for Imlygic, which uses the herpes virus to shrink skin cancers.
But reversing inherited genetic disease remains gene therapy's great promise. And more treatments will reach the market soon. In April, European authorities gave a preliminary green light for a gene therapy for severe combined immune deficiency, to be sold by GlaxoSmithKline. And by 2017, handicappers expect, a Philadelphia company called Spark Therapeutics could win approval in the U.S. for a gene fix that partly reverses one form of blindness. Like the metabolic condition Glybera treats, both these diseases are incredibly rare. Glaxo estimates that only 14 cases of severe combined immune deficiency come to light each year in Europe.
The combination of rare diseases with a costly new technology that needs to be used only once is what could lead to exorbitant prices. Analysts have said Spark’s treatment might cost $500,000 per eye. And with nearly 670 gene-therapy trials under way, and 68 in the later stages, known as Phase III, it’s becoming “urgent” to understand how these therapies will be paid for, says Morrie Ruffin, a consultant with the Alliance for Regenerative Medicine, a trade group. The current system “was not established with these types of products in mind,” he says.
Executives at Glaxo say they don’t expect to charge anything near a million dollars. But with $35 billion a year in revenue, they don’t need to rely on gene therapy for profits. “From a pure business, money-making perspective, it’s a challenge,” says Sven Kili, head of gene-therapy development at Glaxo. “It’s a single treatment that lasts indefinitely and patients don’t come back. And there aren’t many of them. It is not something that would make a venture capitalist jump with joy.” He says Glaxo is thinking of a price “way below” what people might expect for once-in-a-lifetime therapy and “nowhere near” that of Glybera.