Horizon pharma announces health canada approval of procysbi™ to treat nephropathic cystinosis – biopharma global

Horizon Pharma plc, a biopharmaceutical company centered on improving patients’ lives by identifying, developing, obtaining and commercializing differentiated and accessible medicines that address unmet medical needs, and it is affiliate Horizon Therapeutics Canada, today announced that Health Canada has issued a Notice of Compliance (NOC) for PROCYSBI™ (cysteamine delayed-release capsules) for that treatment of nephropathic cystinosis in children and adults 2 years old and older.

“For children diagnosed with nephropathic cystinosis, treatment with cystine-depleting therapy ought to be began as quickly as possible to prevent the intense and potentially existence-threatening effect on organs and tissues,Inches stated Durhane Wong-Rieger, president and ceo, Canadian Organization for Rare Disorders (CORD). “Prior to PROCYSBI there wasn’t any approved therapy in Canada for treating cystinosis. We’re especially pleased that Health Canada grants very timely approval of PROCYSBI, the delayed-release type of the treatment, which supports assure patients avoid toxic build-from cystine within the cells.”

PROCYSBI may be the only cystine-depleting agent approved in Canada for that treatment of nephropathic cystinosis. It’s a delayed-release type of cysteamine bitartrate that actually works by continuously lowering the toxic power of cystine within the cells to limit or avoid the damage an excessive amount of cystine may cause to cells, tissue and organs.

“The Cystinosis Research Foundation is proud to possess funded every bench and early medical trial that brought towards the discovery of PROCYSBI,” stated Nancy Stack, chair from the board, Cystinosis Research Foundation. “The approval of PROCYSBI in Canada is really a milestone achievement for that Canadian cystinosis community.”

Click the link to see Full Article on Check Orphan

Focusing on relief from an uncommon disease? Need Breakthrough Therapy Designation, Steps For Success Designation, RMAT Designation, Food and drug administration or EMA Orphan Designations? The BioPharma Global Regulatory Team might help – We’re the #1 US based not-for-profit Global regulatory matters firm with cost-effective, time-efficient methods to chaperone the journey through the regulatory matters process. Call us at http://www.biopharmaglobal.com/contact/ or by telephone +1(202)660-1826

Resourse: http://biopharmaglobal.com/2017/06/23/horizon-pharma-announces-health-canada-approval-procysbi-treatment-nephropathic-cystinosis/