Raptor Pharmaceutical Corp. announced that it submitted a New Drug Application to the U.S. Food and Drug Administration (FDA) seeking approval to market its investigational drug candidate, cysteamine bitartrate delayed-release capsules (RP103), for the potential treatment of nephropathic cystinosis. In its application, Raptor has requested Priority Review of the NDA, which, if granted, could lead to a decision for marketing approval from the FDA of RP103 for the potential treatment of nephropathic cystinosis in the fourth calendar quarter of 2012. Earlier this week, on March 26, Raptor announced European Medicines Agency (EMA) validation of its Marketing Authorization Application for RP103 for the potential treatment of nephropathic cystinosis.
“A company’s first NDA submission is a significant milestone and we are proud of everyone who helped us to achieve this goal,” said Christopher M. Starr, PhD, Raptor’s Chief Executive Officer. “We could not be more pleased to have now submitted both our NDA, as well as our MAA to the EMA, of RP103 for the potential treatment of cystinosis, by taking advantage of the newly recommended electronic Common Technical Document (eCTD) format.”
The NDA submission for marketing approval includes previously announced data from Raptor’s Phase 3 clinical trial of RP103. 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.
Patrice P. Rioux, MD, PhD, Raptor’s Chief Medical Officer, said, “We believe the positive clinical data generated by our Phase 3 clinical trial demonstrates that RP103 is safe and effective in cystinosis patients. We look forward to working closely with FDA in their review of our application.”
Date: March 30, 2012
Source: Raptor Pharmaceutical Corp.
Filed Under: Drug Discovery