Ocugen’s Repurposed Drug Gets Orphan Designation for Ocular Graft Versus Host Disease

August 11, 2017

Ocugen received an Orphan Drug designation for OCU300 (brimonidine tartrate) for the treatment of ocular graft versus host disease, a common complication in 40 to 60 percent of patients that have undergone allergenic hematological stem cell transplantation or bone marrow transplants, leading to visual impairment.

It is the first-ever orphan designation in this indication, and has no approved treatments, the company said, which is planning a Phase III trial in the near future.

OCU300 is a re-purposed drug — brimonidine tartrate is a glaucoma medication also known as Alphagan. A post-hoc analysis from an exploratory observational study saw beneficial effects in approximately 90 percent of patients without significant side effects, according to Ocugen.

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