FDA Added Unexpected Twist to Dampen Controversy over a Drug That Costs $375,000

The FDA has approved a medication called Ruzurgi developed by Jacobus Pharmaceutical for treating a neuromuscular disorder.

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FDA Twist to Dampen Controversy over Drug Costs $375,000

The U.S. Food and Drug Administration (FDA) made a crafty move to a controversy over a rare-disease drug that became a victim of high-drug price earlier this year.

In what could be a very surprising move, the FDA approved a medicine developed by Jacobus Pharmaceuticals for treating a neuromuscular disorder known as Lambert-Eaton Myasthenic Syndrome (LEMS) for children above six years. Jacobus is a small, family-run pharma company.

Why did the FDA take such a step?

The FDA approved Firdapse, a drug developed by Catalyst Pharmaceuticals, for the treatment of LEMS. Until then, for years, some LEMS patients were able to get the Jacobus drug (Ruzurgi) free under a compassionate program approved by the FDA. However, with the FDA nod, Catalyst got seven years of market exclusivity, which meant Jacobus could no longer market its version of Firdapse.

This FDA’s move created a tumult because Catalyst decided to charge $375,000 for Firdapse. In February, U.S. Senator Bernie Sanders accused the Catalyst of “corporate greed” and “immoral exploitation.” In fact, he asked the FDA to allow Jacobus and other compounders to resume their supplies.

The agency appeared to have heard the call. However, Jacobus has not yet disclosed the price of Ruzurgi.

However, by approving the drug for children, the FDA is allowing doctors to prescribe Ruzurgi for any patient, irrespective of age, because they can prescribe medicines for unapproved or off-label uses.

Duke University researcher Dr. Donald Sanders, who worked with the Jacobus family, said, “If it’s on the market for children, it can be prescribed for adults. I don’t know of any drug that is approved for adults with LEMS other than Firdapse, but we already use many other medicines off label to treat LEMS. This sounds like a workaround.”

A spokeswoman of the FDA said, “The decision to treat a patient with a drug for an unapproved use is up to the treating health care professional and generally speaking, the practice of medicine is not regulated by the FDA. Good medical practice and the best interests of the patient require that physicians use legally available drugs, biologics, and devices according to their best knowledge and judgment.”

However, there are a few extenuating factors. For instance, doctors may prescribe the Jacobus drug, but that does not mean an insurer will automatically provide coverage. Sometimes, insurance companies do not cover off-label use. although in this instance, the price of the Catalyst drug may provide some impetus — if the Jacobus drug costs significantly less.

Laura Jacobus, who runs Jacobus Pharmaceuticals, said a final decision has not been made yet. She said, “I haven’t given it much thought. This decision just came in, but obviously, it will be less than what they’re charging.” She added, “But we’re carrying over the last 27 years the research, development, the compassionate use manufacturing. We’re probably $60 million in the hole. And the post-approval commitments are probably [going to cost] $10 million to $20 million.”