‘A loss for the rest of us’: An FDA approval is a boon for a drug maker, but could come at a major cost for patients
Vickie Moored can walk again. Her words don’t slur, her vision isn’t blurred, and she no longer collapses every day.
The 65-year-old from Elkton, Va., says she has her life back, thanks to the stunning efficacy of a cheap, but unapproved, chemical called 3,4-DAP. It’s a medication she would not have but for the largesse of a tiny, family-run drug maker that has been giving it out, for free, for decades.
But Moored, along with others who share her rare neuromuscular disorder, are concerned that their fortunes could change. The cost of the treatment will likely skyrocket — from next to nothing to potentially more than $100,000 without insurance — now that the Food and Drug Administration has approved a medicine to treat their disease, Lambert-Eaton Myasthenic Syndrome.
“There has been a long-standing need for a treatment for this rare disorder,” the FDA said in a statement this week announcing the approval.
The reality is that physicians have been prescribing 3,4-DAP, a nearly identical treatment, off-label, since the 1980s. It was never formally approved by the FDA; instead, LEMS patients pay about $300 to $500 a month to get the drug from a compounding pharmacy. That, or they’ve been supplied with the drug — for no charge — from the New Jersey-based Jacobus Pharmaceutical Co.
With an FDA approval in hand, however, Florida-based Catalyst Pharmaceuticals now has the ability to charge what could be hundreds of thousands of
