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

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.