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The prices for some drugs have been going up very quickly, and this has gotten a lot of attention from lawmakers and others. And now there is another drug that might soon see its cost spike. But this time, there's a twist. This drug is not widely used, so it has been free for patients who really need it. NPR's Alison Kodjak reports that now a pharmaceutical company wants the rights to the drug and intends to charge quite a hefty price.
ALISON KODJAK, BYLINE: Leigh Shell wasn't all that worried when her teenage daughter, Sarah, started complaining that her legs were tired.
LEIGH SHELL: She began having difficulty climbing stairs, and I just thought that she was - I hate to use the word - lazy, but I didn't understand.
KODJAK: Then, Sarah's eyes stopped moving. She vomited frequently, lost weight, and couldn't concentrate.
SHELL: She lost the feeling in her hands, her feet and her legs.
KODJAK: She ultimately needed a wheelchair. Sarah, who had been a high school cheerleader in her hometown of Gulf Breeze, Fla., was diagnosed with Lambert-Eaton Myasthenic Syndrome, or LEMS. It's a disease where communication between nerves and muscles is blocked. It's rare and debilitating and hard to diagnose. But there is a treatment.
SHELL: When she started taking the Firdapse, it was an instantaneous - within an hour, she's like, mom, I feel like I can skip, which is huge because she can't do that.
KODJAK: Firdapse is the brand name of a drug called 3,4-diaminopyridine, or 3,4-DAP. For the last 20 years, a tiny company called Jacobus Pharmaceuticals had been providing 3,4-DAP to patients for free. The drug is not approved by the FDA and can't be sold with a regular prescription. Company vice president Laura Jacobus says since the market is so small, they never invested in the studies to get it approved. The basic chemicals are widely available, and most people can get the drug through her company or a compounding pharmacy.
LAURA JACOBUS: First and foremost, we wanted to make the drug available to the patients and to the physicians, and that was our number one priority. So that was what we did.
KODJAK: Now a competing company has applied for approval to sell Firdapse as an orphan drug, one that treats a very rare disease. Approval would mean Catalyst Pharmaceuticals gets exclusive rights to sell the medication for seven years. The company tells investors it could make as much as $900 million a year on Firdapse. That suggests it could cost as much as $100 thousand per patient every year. Catalyst CEO Patrick McEnany says those numbers represent a peak opportunity.
PATRICK MCENANY: We've never mentioned a price. We expected we will get whatever, quote-unquote, "orphan drug pricing is."
KODJAK: McEnany says an FDA-approved drug would be safer and easier for patients to get. But doctors who treat LEMS patients aren't convinced.
GORDON SMITH: This is a drug that's cheap to manufacture that's been available for decades.
KODJAK: Gordon Smith specializes in treating LEMS at the University of Utah. He gets 3,4-DAP from Jacobus. He says there will be advantages to a commercially available version of the drug. But, Smith worries that Catalyst will charge too much for something that's not really new.
SMITH: It's akin to trying to patent the latter or rebottle tap water and sell it.
KODJAK: Jacobus Pharmaceuticals is now making its own application to the FDA. Laura Jacobus says the company won't charge near what Catalyst is planning for the drug.
JACOBUS: You know, we've got over 25 years worth of investment in this, and we don't think that it merits that kind of money.
KODJAK: But chances are Catalyst's Firdapse will be approved before even Jacobus finishes its application. And that means patients with Lambert-Eaton or their insurers are likely to see some big bills for medications, perhaps as early as next year. Alison Kodjak, NPR News, Washington.
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