R-Tech Ueno, Ltd. today announced that we have officially received the Orphan Drug Designation from the Ministry of Health, Labour and Welfare (MHLW) for Unoprostone Isopropyl (Unoprostone) (development code : UF-021) for the treatment of retinitis pigmentosa.
Drugs and biologics with orphan drug designation in Japan are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 50,000 patients (maximal incidence of four per ten thousand), with incentives for sponsors to develop orphan products such as priority review of New Drug Applications (NDAs) by the Pharmaceuticals and Medical Devices Agency (PMDA), and grant funding for sponsors.
We are developing a therapeutic agent for treatment of retinitis pigmentosa with unoprostone as the main ingredient and are conducting a phase 3 clinical trial of the ophthalmic solution with unoprostone (development code: UF-021) as the main ingredient (press release of March 13, 2013). A phase 2 clinical trial indicated that the number of patients whose retina sensitivity in the central retina deteriorated was reduced (press releases of June 3 and July 15, 2010). The development of retinitis pigmentosa treatment by unoprostone in Japan has support from government and was adopted in the Adaptable and Seamless Technology Program through Target-Driven R&D (A-STEP) as "Full-scale R&D Stage - Practical Application Type (Contract Development)” by the Japan Science and Technology Agency (JST) (press release of February 1, 2013). We are further increasing our efforts for early approval of this drug.
We licensed the overseas development and commercialization rights for unoprostone to Sucampo Pharmaceuticals, Inc. (Sucampo), for the United States and Canada in 2009, and then for the United States, Canada and the rest of the world excluding our own territories (Japan, South Korea, Taiwan and China) in 2011. While we retain the exclusive rights to manufacture and supply unoprostone products in our territory, we have an efficient partnership with Sucampo for overseas development of unoprostone.
Unoprostone has been designated as an orphan drug for the treatment of retinitis pigmentosa not only by the FDA in the United States but also by the EMA in the European Union, and R-Tech Ueno’s development partner, Sucampo will decide on their path forward assuming our Phase 3 trial is successful.
The above venture will not contribute to any revision of the full-year earnings estimates pre-announced on May 14, 2014.
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