Sarah Shell, a teenager from Gulf Breeze, Florida, has an autoimmune disorder called Lambert-Eaton myasthenic syndrome also known as LEMS. The illness affects about 1,500 to 3,000 people in the U.S., with symptoms often manifesting in young adulthood.

After Shell lost 20 pounds, her legs started feeling tired. Her mother, Leigh, thought her daughter was just being lazy when the latter said she finds it hard to climb the stairs. Sarah's eyes then stopped moving and she would all of a sudden vomit and suffer from migraines. The teenager also found it hard to concentrate, until, finally, she couldn't feel her hands, legs, and feet anymore.

Doctors conducted several tests but no current drug in the market could make Sarah better. Out of options, Sarah began to use Firdapse, an experimental drug used to treat LEMS. The effects were amazing.

"It was instantaneous, within an hour. It was like, 'Mom, I feel like I can skip.' It was a very emotional, tearful moment for us," said Leigh.

Under an orphan drug designation, Firdapse-maker Catalyst Pharmaceuticals applied for the drug's approval from the Food and Drug Administration (FDA). This would give Catalyst Pharmaceuticals a seven-year exclusive right to market Firdapse.

Old Drug, New Drug, Higher Price

The development and approval of LEMS drug Firdapse could be life-saving for patients suffering from the autoimmune disorder. However, many LEMS patients have used the same drug in the past two decades. The "old drug" is called 3,4-diaminopyridine or 3,4-DAP, created by the New Jersey firm Jacobus Pharmaceutical who gives the drug for free.

Via the FDA's compassionate use program, Jacobus Pharmaceutical gave free versions of their experimental drug 3,4-DAP to approximately 600 patients with LEMS. The FDA program enables such experimental medications to become available to patients even if they're not involved in any clinical trials. Such experimental drugs could have life-saving effects, thus the program's existence.

If Catalyst Pharmaceuticals succeeds in getting approval for their version, the drug's price could surge to great heights. Doctors are also worried that an FDA approval of the Firdapse drug could make it harder for some patients to get either version.

In LEMS patients, the communication between the muscles and the nerves are blocked. While there is nothing abnormal about the muscle or the nerve, the blockage between them means no signal is coming through. Doctors said LEMS is a devastating and rare illness that is often hard to diagnose.

In the late 1980s, Jacobus Pharmaceutical started their 3,4-DAP research following the university physician's requests to find a cure for LEMS patients. In early 1990s, the university doctors procured an FDA "Investigational New Drug" permit that allows them to prescribe the drug. Jacobus has been providing doctors the drug ever since. Jacobus offers the 3,4-DAP drug for free. Patients are only charged for a postage stamp that will ship the coolers back to the pharmaceutical company.

Owner Laura Jacobus said their small, family-owned pharmaceutical company doesn't have the big budget or the shareholders to get an FDA approval to sell their experimental drug. Laura highlighted that getting the drug to the patients and doctors was their first priority.

Another firm, BioMarin, pursued the road to gain a full approval from the FDA for their version of the LEMS drug. Catalyst Pharmaceuticals bought Firdapse's rights from BioMarin in 2012. Catalyst CEO Patrick McEnany said making Firdapse available via a usual physician's prescription will benefit many to patients.

"Patients come to us and say 'I have LEMS and I can't get the drug.' Patients don't have access. It's very important that this drug get approved," said McEnany.

Photo: Taki Steve | Flickr

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