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I met Tony in 2001 when he was sent to me with severe muscle weakness. Confined to a wheelchair and unable to care for himself, Tony had been told he had amyotrophic lateral sclerosis, or ALS, a fatal neurodegenerative disorder. It turned out Tony actually had Lambert-Eaton myasthenic syndrome, a rare but treatable neuromuscular disorder. After starting the drug 3,4 diaminopyridine (3,4 DAP), he regained most of his strength and, significantly, his ability to walk and care for himself.
For 78-year-old Tony, 3,4 DAP — which can be obtained at low cost from compounding pharmacies or for free through a compassionate-use program supported by a small privately held company, Jacobus Pharmaceuticals — is a lifesaver. But for Catalyst, the pharmaceutical company that purchased the North American rights to a slightly modified formulation of 3,4 DAP (Firdapse) in 2012, the drug represented a market opportunity to the tune of $300 million to $900 million annually ($60,000 to $100,000 or more per patient).
Although the FDA found Catalyst’s application insufficient and requested additional studies, approval remains possible. Tony and other patients like him live in fear that they will no longer be able to afford the treatment that has returned their quality...