Raptor Pharmaceutical Corp. a biopharmaceutical company focused on developing and commercializing life-altering therapeutics, has received the European Commission (EC) approval for 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).
The approval of the marketing authorization application (MAA) by the EC follows the positive recommendation by the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) in June 2013. The orphan medicinal product designation provides a 10-year period of market exclusivity in the EU that starts from the notification date of the EC Decision granting the MAA.
"The EC approval of Procysbi brings us a major step closer to providing access to this important new therapeutic option to cystinosis patients in Europe," said Christopher M. Starr, Raptor's chief executive officer. "Active dialogue has begun with a number of EU member states initially as part of a phased introduction of Procysbi to patients with this debilitating disease."
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. Procysbi is taken orally every twelve hours. Procysbi was engineered to bypass absorption in the stomach with an extended terminal half-life so that patients experience steady drug levels in their bodies for the full 12-hour dosing period. Randomized controlled clinical trials and extended treatment with Procysbi therapy demonstrated consistent and continuous control of white blood cell cystine levels.
The EC approval of Procysbi was based on data from six clinical trials, including a multi-center randomized, active-controlled phase 3 trial of 43 patients with nephropathic cystinosis and extension data from that trial. The EC's Committee for Medicinal Products for Human Use (CHMP) concluded that the submitted study data support clinical efficacy of Procysbi similar to that of immediate-release cysteamine with a dosing schedule that may promote a better compliance.