Osaka, JAPAN (March 2, 2015) -- Santen Pharmaceutical Co., Ltd. announced that the European Medicines Agency (EMA) has accepted the company's Marketing Authorization Application (MAA) filing for the use of intravitreal sirolimus, an investigational mTOR inhibitor, for the treatment of noninfectious uveitis (NIU) of the posterior segment. The MAA filing marks the beginning of a regulatory review process for intravitreal sirolimus, which has the potential to address an unmet need in the European Union (EU) where NIU of the posterior segment is a leading cause of blindness1.
"Given the risks associated with currently available therapeutic options for NIU of the posterior segment, a chronic inflammatory condition in many cases, there is a significant need for novel treatments that could be used in a sustained manner to optimize clinical outcomes," says Dr. Naveed Shams, Chief Scientific Officer and Head, Global R&D, Santen Ltd. "If approved, intravitreal sirolimus would represent a significant step forward in the management of NIU of the posterior segment of the eye."
The EMA submission is supported by data from SAKURA (Study Assessing double‐masKed Uveitis tReAtment), a pivotal Phase 3 study. The submission seeks approval to market the sirolimus, 440 µg dose for the chronic treatment of NIU of the posterior segment of the eye. Key outcomes supporting the indication include data on proportion of subjects achieving a vitreous haze score of 0 at Month 5, proportion of subjects achieving a vitreous haze score of 0 or 0.5+ at month 5, the proportion of subjects achieving an improvement in vitreous haze score of 2 units, and proportion of subjects successfully tapering-off systemic corticosteroids at Month 5.
Intravitreal sirolimus, a first-in-class local immunoregulatory therapy, being evaluated for the treatment of NIU of the posterior segment, inhibits mTOR, which plays a critical role in stimulating T-cell proliferation leading to the release of proinflammatory cytokines. By inhibiting mTOR, sirolimus interrupts a critical pathway that perpetuates the inflammatory process, controlling the disease's progression. Intravitreal sirolimus is a proprietary ocular formulation of sirolimus currently under review by the EMA.
Uveitis is an intraocular inflammatory condition, which may or may not have an infectious component, and is often classified by anatomic location in the eye. NIU of the posterior segment includes intermediate uveitis which can affect the ciliary body and vitreous; posterior uveitis which can involve the vitreous, choroid, retina, and/or optic nerve; and panuveitis which encompasses anterior and posterior segments of the eye. While currently available treatments, such as systemic corticosteroids and immunomodulatory therapies, are effective in treating inflammation in the eye, they are associated with a range of serious adverse effects that may limit the ability to achieve long-term therapeutic success.
Santen Pharmaceutical Co., Ltd., is a billion dollar global company headquartered in Osaka, Japan since 1890. With subsidiaries in the U.S., Europe and Asia, Santen researches, develops and markets ophthalmic products for physicians worldwide. Santen's global product pipeline includes innovative compounds in all stages of clinical development. Among ophthalmic pharmaceutical companies, Santen holds the top share within the Japanese market, is a market leader in China, and is one of the leading ophthalmic companies worldwide. Additional corporate information is available online at www.santeninc.com.
Information provided in this press release contains so-called "Forward-looking Statements". The realizations of these forecasts are subject to risk and uncertainty from various sources. Therefore, please note that the actual results may differ significantly from the forecasts. Business performance and financial condition are subject to the effects of change in regulations made by the governments of Japan and other nations concerning medical insurance, drug pricing and other systems, and to fluctuations in market variables such as interest rates and foreign exchange rates.
This press release is also prepared in other languages. However this English document is the original document, and documents prepared in other languages are for your reference only. In case of any discrepancy between the English and translated versions, the English version will prevail. Please refer to http://www.santen.com for the original English document
Nicolas Merigeau / Emmanuel Huynh
Tél. : +33-1-4471-9498
Directrice de la Communication - Santen Europe
Tél. : +33-6-6554-6019
General Manager, Corporate Communications Group
Santen Pharmaceutical Co., Ltd
1. Wakefield D, Chang JH. Epidemiology of uveitis. Int Ophthalmol Clin. 2005;45(2):1-13.