| Sumario: | Cysteamine (Procysbi), a delayed-release formulation containing 25 mg or 75 mg of cysteamine bitartrate (referred to as delayed-release cysteamine) was approved by Health Canada for the treatment of patients with nephropathic cystinosis. Cystinosis is a recessive autosomal genetic ultra-rare disease affecting 1 in 100,000 to 200,000 live births in Europe (1 in 62,500 in Saguenay-Lac-St-Jean, Quebec), characterized by renal and extrarenal (i.e., diabetes, osteopenia, muscular weakness and wasting, growth retardation, etc.) complications. Cysteamine acts through preventing or delaying the occurrence of renal and extrarenal complications and hence prolonging life expectancy. The recommended dose of delayed-release cysteamine is 1.3 g/m2 daily to be given in two equal doses per day. At the manufacturer's submitted price (11.30 per 25 mg capsule, 33.89 per 75 mg capsule), the daily dose cost will vary from 372.90 in a two-year-old child (based on a body surface area of 0.63 m2) to 881.40 in adults (based on a body surface area of 1.50 m2), or 136,000 to 321,000 per patient annually.
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