Raptor Pharmaceutical Corp. Announces EMA Validation of Marketing Authorization Application for RP103 for the Treatment of Nephropathic Cystinosis

Raptor Pharmaceutical Corp. RPTP, announced that the European Medicines Agency has determined that Raptor's Marketing Authorization Application (MAA) for its investigational drug candidate, RP103 for the potential treatment of nephropathic cystinosis, submitted earlier this month, is valid. Validation of the MAA confirms that the submission is sufficiently complete to begin the formal review process. "This filing represents the Company's first registration application and a significant milestone in our journey to make RP103 available to cystinosis patients throughout the world," said Christopher M. Starr, Ph.D., Raptor's Chief Executive Officer. "As we move closer to potential approvals, we remain dedicated to serving the cystinosis patients, medical community and foundations and thank them for their critical roles in bringing us to this point in the program." The MAA submission for registration includes data from Raptor's Phase 3 clinical trial of RP103 (cysteamine bitartrate gastro-resistant capsules), which was announced in July 2011. As reported, RP103 met the clinical trial's sole primary endpoint and there were no unexpected serious safety concerns attributable to RP103 experienced by patients in the trial. The trial was conducted at three clinical sites in the U.S. and five clinical sites in Europe.