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Some patients say they ve lost out in unusual battle over rare disease drug

"This has been nothing but a circus,” said Eric Sorenson, a Mayo Clinic neurologist who treats patients with the rare neuromuscular disease.

Appeals court rules FDA capriciously approved rare disease drug

Appeals court rules FDA capriciously approved rare disease drug
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Refractory Angina Market to Increase at a 7 97% CAGR During the Study Period [2018-2030], Speculates DelveInsight

Refractory Angina Market to Increase at a 7 97% CAGR During the Study Period [2018-2030], Speculates DelveInsight
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Catalyst wins an unexpected victory in Canada over a rare disease drug

DON EMMERT/AFP via Getty Images In an unexpected move, Canada’s Federal Court quashed the approval of a rare disease drug after the manufacturer of a rival medicine claimed that Health Canada had originally issued an “incorrect and unreasonable” endorsement. In a 63-page ruling, Justice Martine St-Louis set aside the approval issued by the regulator last year. As a result, the government will now have to review the marketing file submitted by Jacobus Pharmaceuticals, a small, family-run company that sells a drug to treat people with a rare neuromuscular disorder called Lambert-Eaton myasthenic syndrome, or LEMS. A competitor, Catalyst Pharmaceuticals (CPRX), is seeking to push back the approval until 2028. We asked Jacobus and Health Canada for comment and will update you accordingly.

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