EU approves Procysbi for Nephropathic Cystinosis
Raptor Pharmaceutical Corp. announced that the European Commission has approved Procysbi, gastro-resistant hard capsules of cysteamine (as mercaptamine bitartrate) as an orphan medicinal product for the treatment of proven Nephropathic Cystinosis for marketing in the European Union (EU). Procysbi is indicated for the treatment of proven Nephropathic Cystinosis.
Cysteamine reduces cystine accumulation in some cells (e.g. leukocytes, muscle and liver cells) of Nephropathic Cystinosis patients and, when treatment is started early, it delays the development of renal failure.
The EC approval of Procysbi was based on data from six clinical trials, including a multi-center randomized, active-controlled Phase III trial of 43 patients with Nephropathic Cystinosis and extension data from that trial.
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