This article is more than 5 years old. Information may no longer be current.

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

Add topic to email alerts

Receive an email when new articles are posted on

Please provide your email address to receive an email when new articles are posted on .

Subscribe

Added to email alerts

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

Click Here to Manage Email Alerts

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

Click Here to Manage Email Alerts

Back to Healio

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

Back to Healio

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.