Catching Catalyst Pharma by surprise, FDA approves Jacobus LEMS drug for pediatric patients — based on adult data
When Catalyst Pharmaceuticals’ drug for Lambert-Eaton myasthenic syndrome (LEMS) — a rare, autoimmune disorder that affects the connection between nerves and muscles — was approved last year, carrying a $375,000 annual price tag, some patients were not exactly thrilled. Hundreds of patients had been able to access a similar drug from compounding pharmacies for a fraction of the cost, or from a small, family-run New Jersey-based company called Jacobus Pharmaceuticals for free, as part of an FDA-ratified compassionate use program.
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