The FDA has granted orphan drug designation for OCU300 to treat ocular graft-versus-host disease, according to a press release from Ocugen.
Ocular graft-versus-host disease affects 40% to 60% of patients who have undergone allergenic hematological stem cell or bone marrow transplants, and it induces severe ocular surface disease, the release said.
OCU300 (brimonidine tartrate) showed a beneficial effect in 90% of patients treated with it, according to a post hoc analysis of an exploratory observational study.
This is the first orphan drug designation by the FDA for ocular graft-versus-host disease, Shankar Musunuri, PhD, MBA, Ocugen’s chairman, CEO and co-founder, said in the release. He also said the company plans to advance the clinical development of OCU300, with a proprietary nanoemulsion, into a phase 3 clinical trial.