FDA grants Priority Review for Repligen's SecreFlo NDA

Repligen Corporation (NASDAQ:RGEN) announced today that the U.S. Food and Drug Administration (FDA) has accepted for filing and granted Priority Review to the Company's new drug application (NDA) for SecreFlo™ for the improved detection of pancreatic duct abnormalities in patients with pancreatitis. Under the Prescription Drug User Fee Act (PDUFA), the FDA's goal for completing a Priority Review and delivering a decision on marketing approval is reduced to six months, compared to ten months for a Standard Review. The FDA has assigned a PDUFA goal date of June 21, 2012 to the SecreFlo™ NDA.

“The commitment made by the FDA to expedite the review of the SecreFlo™ NDA underscores the need for safer, noninvasive methods for physicians to evaluate their patients with pancreatitis”

Priority Review designation is given to product candidates that offer a significant improvement in the treatment, diagnosis or prevention of a disease or that address an unmet medical need. Priority Review for the SecreFlo™ NDA is based on the FDA's recognition of the need for safer, noninvasive alternatives to the diagnostic use of endoscopic retrograde cholangiopancreatography (ERCP). ERCP is an invasive endoscopic procedure that has traditionally been used to examine and evaluate diseases of the pancreas. More recently, the medical community has recognized the need for a safer diagnostic approach based on documented morbidity and mortality risks associated with the diagnostic use of ERCP and current practice recommendations issued by the National Institutes of Health (NIH). Magnetic resonance imaging (MRI) offers a safer and noninvasive alternative to diagnostic ERCP; however, its utility is currently limited by the lack of an approved agent to enhance MRI images of the pancreatic ducts in order to achieve a definitive diagnosis. SecreFlo™, a synthetic version of the human hormone secretin, when used in combination with MRI, may fill this unmet need. There are approximately 300,000 abdominal MRI procedures conducted in the U.S. and Europe each year that may directly benefit from the addition of SecreFlo™.

"The commitment made by the FDA to expedite the review of the SecreFlo™ NDA underscores the need for safer, noninvasive methods for physicians to evaluate their patients with pancreatitis," said Walter C. Herlihy, Ph.D., President and Chief Executive Officer of Repligen. "If approved, we believe SecreFlo™ has the potential to improve pancreatic imaging and reduce the need for diagnostic endoscopy, to the benefit of patients and payers."

Clinical Basis for SecreFlo™ NDA

The SecreFlo™ NDA was submitted to the FDA based on positive outcomes from the re-read of the Company's Phase 3 clinical study. This pivotal study was a multi-center, baseline controlled, single dose study in which 258 patients were enrolled at 23 clinical sites in the U.S. and Canada. Each patient in the study received an MRI of the pancreas with and without SecreFlo™ and separately underwent endoscopy (ERCP) as a diagnostic reference. The MRI images were randomized and independently read and reviewed by three central radiologists.

The Phase 3 study re-read demonstrated that all endpoints were met with statistical significance and that SecreFlo™ was safe and well tolerated. The study's co-primary endpoints were achievement of a statistically significant improvement in sensitivity of detection of abnormalities of the pancreatic ducts with minimal loss in specificity by two of the three central radiologists reading the MRI images. The study met its primary endpoints, demonstrating that the addition of SecreFlo™ to MRI resulted in a highly statistically significant improvement (p<0.0001) in sensitivity of detection of ten prespecified abnormalities of the pancreatic ducts, with minimal loss in specificity (<7.5%). All three secondary endpoints were also met, with each demonstrating highly statistically significant improvements (p<0.0001) in image quality, visualization of the main pancreatic duct and diagnostic confidence when compared to MRI alone. A radiologist's ability to more confidently identify and characterize pancreatic duct abnormalities has the potential to substantially improve "next-step" clinical decisions (patient triage) and patient outcomes.

SecreFlo™ was previously granted Fast Track designation by the FDA and also has Orphan Drug status, which qualifies Repligen to receive seven years of U.S. marketing exclusivity for SecreFlo™, if approved. In addition to it's U.S. application for approval of SecreFlo™, the Company plans to file a marketing authorization application (MAA) during the first quarter of 2012 for potential approval of SecreFlo™ in the EU.