CHMP recommends Procysbi (Raptor Pharma) to treat Nephropathic Cystinosis

On 27 June 2013, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a marketing authorisation for the medicinal product Procysbi, 25 mg, 75 mg, gastro-resistant capsules, hard intended for the treatment of Nephropathic Cystinosis. Procysbi was designated as an orphan medicinal product on 20 September 2010. The applicant for this medicinal product is Raptor Pharmaceuticals Europe B.V.

The active substance of Procysbi is mercaptamine, an �other alimentary tract and metabolism� product (ATC Code: A16AA04). Mercaptamine participates within lysosomes in a thiol disulfide interchange reaction converting cystine into cysteine and cysteine-cysteamine mixed disulfide, both of which can exit the lysosome in patients with Cystinosis. The benefits with Procysbi are its ability to reduce 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.