FDA undercuts $375,000 drug in surprise move

The US Food and Drug Administration created a workaround this week that effectively undercuts the $375,000 price tag of a drug that became the poster child for concerns about the pharmaceutical industry.

The FDA announced Monday that it was approving a separate drug for pediatric patients with a rare, painful neuromuscular disease called Lambert-Eaton myasthenia syndrome, also known as LEMS. The disease weakens and fatigues muscles, causing agonizing pain to the point at which patients struggle to walk.

The move sparked celebration in the LEMS community and tanked the stock of Catalyst Pharmaceuticals, maker of the $375,000 drug, called Firdapse. The company’s stock price has dropped about 50% since Monday.

The FDA said it was approving Ruzurgi — made by a small company called Jacobus Pharmaceutical Co. — for children with LEMS, ages 6 to 17.

“We continue to be committed to facilitating the development and approval of treatments for rare diseases, particularly those in children,” said Dr. Billy Dunn, director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research.

“This approval will provide a much-needed treatment option for pediatric patients with LEMS who have significant weakness and fatigue that can often cause great difficulties with daily activities.”

Although Ruzurgi was approved for pediatric patients, the FDA decision makes it possible for adults with LEMS to get the drug off-label if their treating physician believes that it will help them.

“Generally, if a drug is FDA-approved, it can be used off-label as long as physicians feel it is indicated for the patient,” said Dr. Vincent Rajkumar, a hematologist oncologist with the Mayo Clinic. “That’s how drugs approved for one disease can be used for almost any other.”

Jacobus has not announced a price for Ruzurgi, but owner Laura Jacobus said the company wants to make sure the drug is affordable for patients.

“We have been working on this drug for a long time and have invested significant resources,” she said in a written statement. “Although we have not established a price for Ruzurgi, it remains our goal to ensure that Ruzurgi is affordable and available to pediatric patients living with LEMS.”

The FDA said it is not known how many pediatric LEMS patients there are, but Jacobus estimates that there are fewer than 15. According to the National Organization for Rare Disorders, LEMS affects about 400 people in the United States. The FDA said that overall, 3 people out of 1 million worldwide have LEMS.