Health Care

New drug could give JoCo man with rare condition an alternative to $375,000 medicine

Will Schuller and his family have been learning a lot about “off-label” prescribing lately.

“Off-label” is when a doctor prescribes a drug for something other than its Food and Drug Administration-approved purpose. And it may now give Schuller an alternative to a life-saving medication with a list price of almost $400,000 a year.

“It’s definitely something that kind of came unexpectedly,” said Schuller. “Out of the blue, but good news obviously.”

Schuller, 22, of Overland Park, has an extremely rare autoimmune condition called Lambert Eaton-Myasthentic Syndrome, or LEMS. Its symptoms first reared up when he was a senior at Blue Valley Northwest and quickly became debilitating, consigning the avid jogger to a wheelchair within months.

He recovered just as speedily, though, once he was properly diagnosed and got access to an experimental medication produced by a small company in New Jersey called Jacobus Pharmaceutical.

Jacobus had not gone through the expensive and time-consuming process of seeking FDA approval to sell the drug, and was instead distributing it to hundreds of patients like Schuller for nothing but the cost of shipping.

Then another company, Florida-based Catalyst Pharmaceuticals, got a slightly modified version through the FDA pipeline using the Orphan Drug Act, a law meant to encourage companies to develop innovative new drugs for rare conditions.

The law essentially barred any other companies, including Jacobus, from distributing competing products for seven years. With no competition, Catalyst set a list price of $375,000 a year — or more than $30,000 for each monthly prescription for the average dose of the drug, Firdapse.

Doctors, patient advocates and U.S. Sen. Bernie Sanders accused Catalyst of exploiting a loophole in the Orphan Drug Act to make big money on a product that wasn’t all that innovative.

But now the FDA has given patients a potential workaround, and Catalyst a potential competitor.

Firdapse was approved only for adults. Last week, the FDA approved Jacobus’ drug, Ruzurgi, for kids age 6 to 17, even though much of the data came from trials on adults. That means doctors will be able to prescribe Ruzurgi, off-label, for LEMS patients of any age.

“There’s people older than 17 on the (LEMS) Facebook page saying they’ve already talked to their neurologist about getting it and they seem to think there’s going to be no trouble,” Schuller said.

“Everybody is very excited about it,” said Schuller’s mom, Ann.

Well, maybe not everybody. Catalyst’s stock plunged almost 40% on news of Ruzurgi’s approval.

The company did not respond to a request for comment.

The Schullers are now anxiously awaiting news of Ruzurgi’s price. Jacobus co-owner Laura Jacobus told STAT, a medical news website, that it would be less than Firdapse, but also said her company has at least $60 million in research, development and charitable donations of the drug over 27 years to make up.

There’s also a chance that Catalyst will mount a legal challenge.

In the mean time, Will Schuller is covered under his parents’ Blue Cross and Blue Shield of Kansas City plan and pays only about $10 a month out of pocket for Firdapse. But his insurer pays about $40,000 a month, Schuller said, because he takes a higher-than-average dose.

He and his mom both said they expect those costs to be passed on to consumers.

“Our premiums will probably eventually go up,” Schuller said.

He also won’t be on his parents’ insurance forever. He just graduated from Tulsa University and will soon take a job at Honeywell. When he checks out the company’s health insurance offerings, he plans to read the prescription drug coverage section very carefully.

“That’s the pending question for my future,” Schuller said. “What insurance plan is the company going to provide me?”

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Kansas City Star health reporter Andy Marso was part of a Pulitzer Prize-finalist team at The Star and previously won state and regional awards at the Topeka Capital-Journal and Kansas Health Institute News Service. He has written two books, including one about his near-fatal bout with meningitis.