Orphan drug designation for uveitis treatment approved

Santen announced the FDA approval of the orphan drug designation of sirolimus, a uveitis treatment.

The FDA approval follows the European Commission's decision to grant sirolimus orphan drug status in September 2011. Sirolimus, otherwise known as rapamycin, is used as an immunosuppressive and antiproliferative agent for the treatment of chronic/refractory anterior non-infectious uveitis, intermediate uveitis, panuveitis and uveitis affecting the posterior segment of the eye.

Currently, sirolimus is being assessed in the Phase III study SAKURA (Study Assessing Double-Masked Uveitis Treatment). The investigation will be used to determine the safety and efficacy of various sirolimus doses used to treat non-infectious posterior uveitis.

Dr Toshiaki Nishihata, Head of Research and Development Division, said, "This is an important milestone in the development of sirolimus for non-infectious uveitis affecting the posterior segment of the eye."