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Catalyst Pharma: Orphan Drug Poseur, Profiteer

Why supply 3,4-Dap for free? Why not get the drug approved and charge patients for access?

"Because supplying the drug for free is the right thing to do," said Laura Jacobus. "My father believed, and we still believe, that we have a moral responsibility to support this fragile patient population and not to profit from them. A lot of people believe the government or the insurance companies will support these patients and pay for their drug but at the end of the day, we all pay for it."

David and Laura Jacobus aren't socialists. They have no problem with companies developing novel drugs to treat disease and making money selling them. But that's not what Catalyst is doing with Firdapse in the U.S. or what Biomarin has done with the drug in Europe, Laura Jacobus says.

"Firdapse is not a new compound. It's the same drug we make. What Catalyst is doing is not the same as a company profiting from a new invention. What Catalyst is doing is making money off LEMS patients. They don't want to help LEMS patients, they just want to make money. If I worked for Catalyst, I wouldn't be able to sleep at night," she says.

Jacobus is working to stop Catalyst from gaining FDA approval for Firdapse. After years of supplying 3,4-Dap for free through compassionate use programs, Jacobus decided to conduct its own clinical trial with the intent of getting the drug formally approved by the FDA.

The Jacobus pivotal study of 3,4-Dap in LEMS patients is underway, competing directly with Catalyst's Firdapse study.

Laura Jacobus wouldn't disclose how many patients are enrolled to date but said enrollment is going "very well" and the study is "getting close to the end." The company's long relationship with U.S. doctors who treat LEMS patients has helped greatly, she added. [The <a href=",4-Diaminopyridine" rel="nofollow">Wikipedia entry for 3,4-Dap</a> even encourages LEMS patients not to enroll in Catalyst's Firdapse study.]
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