Santen Pharmaceutical (Osaka, Japan) and Santen (Emeryville, CA) announced the U.S. Food and Drug Administration (FDA) has issued a complete response letter for the New Drug Application (NDA) for intravitreal (IVT) sirolimus (DE-109). IVT sirolimus is an investigational therapy developed as a potential treatment for adults with noninfectious uveitis of the posterior segment, a leading cause of preventable blindness in working-age adults.
The complete response letter indicates that the FDA is unable to approve the application in its present form and requests additional substantiating evidence to demonstrate efficacy of IVT sirolimus in the treatment of noninfectious uveitis of the posterior segment.
"We are evaluating the FDA's response and will work closely with the agency to determine the best path forward. We strongly believe in the benefits of IVT sirolimus for noninfectious uveitis of the posterior segment patients, and will continue to work towards our goal of bringing a local, non-steroidal therapy to patients suffering from this sight-threatening disease," said Naveed Shams, MD, PhD, Chief Scientific Officer and Head of Global Research and Development of Santen. "Santen remains focused on our mission to develop and provide innovative therapies to patients suffering from ophthalmic diseases in the United States and around the world."
The CRL is not expected to have a material impact on FY2017 earnings forecasts.
IVT sirolimus is being evaluated as a potential treatment for adults with noninfectious uveitis of the posterior segment (intermediate, posterior and panuveitis). IVT sirolimus is a locally-administered, non-steroidal immunoregulator that has been investigated for the resolution of intraocular inflammation associated with noninfectious uveitis of the posterior segment. The New Drug Application for IVT sirolimus was supported by the results from the SAKURA (Sirolimus study Assessing double-masKed Uveitis tReAtment) Program, the largest Phase III global program for noninfectious uveitis of the posterior segment to date. The SAKURA Program included two multi-national, randomized, double-masked, active-controlled studies in adult patients with active non-infectious intermediate, posterior or panuveitis.