Burton-on-Trent, UK – 18 February 2015 – 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 this 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.”
1 Gahl 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.
About Named Patient Programs
Named Patient Programs provide controlled, pre-approved access to unapproved medicines in response to requests from Healthcare Professionals on behalf of specific or ‘named’ patients before those medicines are licensed in the patients’ home country.
About Clinigen Group
The Clinigen Group is a specialty global pharmaceutical company headquartered in the UK, with offices in the US and Japan. The Group, dedicated to delivering ‘the right drug, to the right patient at the right time’, has three operating businesses; Specialty Pharmaceuticals (Clinigen SP), Clinical Trials Supply (Clinigen CTS), and Global Access Programs (Clinigen GAP). Clinigen GAP develops and implements global access programs for biotechnology and pharmaceutical companies and has provided access to unlicensed, licensed and end-of-lifecycle products to thousands of patients. Clinigen has experience in the implementation of more than 30 access programs worldwide.
For more information, please visit www.clinigengroup.com.
Issued for and on behalf of Clinigen Group by Instinctif Partners.
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Melanie Toyne-SewellManaging Partner