August 09, 2017
1 min read

Ocugen’s treatment for ocular graft-versus-host disease gets orphan drug designation

You've successfully added to your alerts. You will receive an email when new content is published.

Click Here to Manage Email Alerts

We were unable to process your request. Please try again later. If you continue to have this issue please contact

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.