OREANDA-NEWS. Clinigen Group plc ('Clinigen' or the 'Group', AIM:CLIN) announces the latest program to be managed by Clinigen Global Access Programs (Clinigen GAP). The program will provide access to Raptor Pharmaceuticals Corp.'s PROCYSBI® (cysteamine bitartrate) for individual patients with nephropathic cystinosis. Through the program PROCYSBI® will be available worldwide, except for the USA where the drug is already commercially available, LATAM and a small number of European territories.

Nephropathic cystinosis is a rare, inherited condition caused by a build up of cystine that affects every cell in the body. Without treatment, patients can suffer from kidney and eye problems and eventually severe and irreversible damage to the heart, central nervous system and muscles. Approximately 2,000 people worldwide are currently diagnosed with the disorder1.

PROCYSBI® is a delayed-release form of cysteamine bitartrate that can be used to manage cystinosis by continually reducing the toxic levels of cystine in the cells. This can help to prevent cellular damage and delay the progression of cystinosis and its complications.

Mark Corbett, Senior Vice President, Clinigen GAP said: "Nephropathic cystinosis is a serious, progressive disease and the initiation of this program for PROCYSBI® is a key step in getting an important new therapeutic option to patients with limited treatment options.  We are very proud that Raptor have selected Clinigen GAP as their partner to manage this global access program. We are able to utilize our extensive international expertise in project management, regulatory advice and logistics in order to ensure Raptor can effectively address this area of unmet medical need."

1Gahl WA, Kuehl EM, Iwata F, Lindblad A, Kaiser-Kupfer MI. Corneal crystals in nephropathic cystinosis: natural history and treatment with cysteamine eyedrops. Mol Genet Metab. 2000;71:100-120.