FDA grants orphan drug status to Ocugen’s OCU300 for oGVHD treatment


The US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) for Ocugen’s OCU300 (brimonidine tartrate) to treat patients with ocular graft versus host disease (oGVHD).

Ocugen is a clinical stage biopharmaceutical company that focuses on the development of new treatments for sight-threatening diseases.

oGVHD is a common complication found in 40% to 60% of patients who have undergone allergenic haematological stem cell transplantation (allo-SCT) or bone marrow transplants.

The disease is driven by autoimmune inflammation and causes serious ocular surface disease, which over time declines the quality of the patient’s life significantly and hinders daily activities due to visual impairment.

"We are very excited to receive the first ever orphan drug designation by the FDA for oGVHD, emphasising the unmet medical need for patients with this disease."

OCU300 is a re-purposed drug that is currently being developed through the FDA's 505(b)(2) pathway for the treatment of oGVHD.

Ocugen chairman, co-founder and CEO Dr Shankar Musunuri said: “We are very excited to receive the first ever orphan drug designation by the FDA for oGVHD, emphasising the unmet medical need for patients with this disease.

“This is a significant milestone that will allow us to further advance the clinical development of OCU300, with a proprietary nanoemulsion, into a phase III clinical trial in the near future.”

A post-hoc analysis of OCU300 conducted in an exploratory observational study showed the treatment’s beneficial effect in nearly 90% of oGVHD patients without causing any significant side effects.

Last year, the biopharmaceutical company entered an exclusive worldwide licence agreement with the University of Illinois for OCU300.