Sale sets remedy’s price tag soaring

100 pills free in ’16, $15,001 now

For four decades, Don Anderson of Seattle has been taking the same drug to help control the temporary bouts of immobility and muscle weakness caused by a rare genetic illness called periodic paralysis.

“It’s like putting a 50-pound pack on your back and standing up at the dinner table,” Anderson, 73, said. “It’s like wearing lead shoes around all the time.”

The drug Anderson has been taking all these years was originally approved in 1958 and used primarily to treat glaucoma under the brand name Daranide, its price so unremarkable that he can’t quite remember how much it cost at the pharmacy counter.

But the price has been on a roller coaster in recent years — zooming from a list price of $50 for a bottle of 100 pills in the early 2000s up to $13,650 in 2015, then plummeting to the point where it was free, before skyrocketing back up to $15,001 after a new company, Strongbridge Biopharma, acquired the drug and relaunched it.

“I’m constantly hearing that public pressure, public shaming will be sufficient to curb these bad actors in these industries. It often feels if you take your atten-

tion off of them, even for a second, they’ll revert to these old ways,” said Rachel Sachs, an associate law professor at Washington University in St. Louis. “It’s just another example of how the system has some problems that need to be fixed.”

The zigzagging trajectory of the price of Daranide, now known as Keveyis, shows just how much freedom drug companies have in pricing therapies — and what a big business opportunity the sale of drugs for extremely rare diseases has become. It also illustrates how well-intentioned policy to help spur the development of “orphan” drugs for very rare diseases can have unintended consequences.

Some people with the rare neuromuscular condition began taking Daranide off-label to help control their disease. With a list price of $50 for 100 pills in 2001, it wasn’t a drug people remember as hard to obtain. (Pricing data were obtained from Truven Health Analytics, part of the IBM Watson Health business.)

In the early 2000s, Daranide was discontinued by Merck. Other glaucoma treatments were available, but a small group of periodic paralysis patients who had found that Daranide controlled their symptoms better than other drugs were left with few options. They found ways to get the drug, importing it from Europe or South Korea. Anderson recalls the expense as about $250 or $300 a month.

In 2008, a family affected by the disease that also owned Taro Pharmaceutical Industries, a generic pharmaceutical company, decided to acquire Daranide from Merck. The goal was to make the drug reliably available to patients at a reasonable cost, Barrie Levitt, the former chairman of the company, and his son Jacob said in 2016.

Jacob suffers from periodic paralysis, and although he took a different drug to control his disease, he became aware from his work in the patient advocacy community that Daranide had been discontinued, forcing patients to look for alternatives or find sources to import. He said Taro spent less than $500,000 to acquire the old drug.

But Taro was taken over by another generic company, Sun Pharmaceutical Industries, in 2010. When the drug was approved in 2015 as a raredisease treatment for periodic paralysis, it got a new name, Keveyis, and a new price: $13,650 for 100 pills. Although Keveyis is actually a decadesold drug, its federal approval for periodic paralysis came with a seven-year period of exclusive marketing rights.

In 2016, after The Washington Post asked questions about the high price of the drug, Sun Pharmaceutical said it would give the drug away for free. The company said the timing was coincidental and reflected the fact that it had made less than $1 million on the drug, not enough to recoup the investment the company had made in marketing and patient support services.

Late last year, Sun Pharmaceutical agreed to sell Keveyis to a biotech company, Strongbridge Biopharma, for $8.5 million. In April, Strongbridge Biopharma relaunched the drug — and in August, it increased the list price from $13,650 to $15,001 for a bottle of 100 pills.

In a PowerPoint presentation for investors, Strongbridge Biopharma estimated that the annual price of treatment for Keveyis would range from $109,500 to $219,000, depending on the dosage the patient took. One slide shows that the drug is covered broadly by insurers.

In November, the company announced $2.5 million in sales over the most recent quarter — a 67 percent increase over the previous quarter’s $1.5 million in sales. Strongbridge Biopharma said it would expand its sales force, and executives said in a conference call that the company’s medical affairs team had met with 75 medical leaders and was training speakers to lead “peer-to-peer educational programs.”

Lindsay Rocco, a spokesman for Strongbridge Biopharma, declined to answer questions about why the company increased the price of the drug earlier this year. Instead, she issued a company statement saying that periodic paralysis affected only 5,000 people in the United States and that the drug could provide benefits for those people.

“Strongbridge is committed to serving the unmet needs of the primary periodic paralysis and other rare-disease communities,” the statement said.

Sun Pharmaceutical did not answer questions about why the company sold the drug after dropping the price to zero.