Ocugen Receives FDA Ocugen Receives FDA Orphan Drug Designation for OCU300 for Treatment of oGVHD De

Ocugen announced the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) for OCU300 (brimonidine tartrate) for the treatment of ocular graft versus host disease (oGVHD). Ocular GVHD is a common complication that occurs in 40-60 percent of patients who have undergone allergenic hematological stem cell transplantation (allo-SCT) or bone marrow transplants. Driven by autoimmune inflammation, oGVHD induces severe ocular surface disease, which over time significantly diminishes quality of life, and restricts daily activities due to visual impairment.

"We are very excited to receive the first ever orphan drug designation by the FDA for oGVHD, emphasizing the unmet medical need for patients with this disease," said Shankar Musunuri, PhD, MBA, chairman, CEO and co-founder of Ocugen. "This is a significant milestone that will allow us to further advance the clinical development of OCU300, with a proprietary nanoemulsion, into a phase 3 clinical trial in the near future."

Ocugen executed an exclusive worldwide license agreement for OCU300 with the University of Illinois at Chicago last year.

"I'm pleased that Ocugen has received orphan drug designation for oGVHD," Dr. Sandeep Jain, Director of the ocular GVHD Clinic at the University of Illinois at Chicago and an inventor of OCU300 for the treatment of oGVHD, said. "This is a debilitating condition for which no approved pharmaceutical therapeutics exist."

The FDA Office of Orphan Products Development (OOPD) grants orphan designation for novel drugs or biological that treat a rare disease or condition affecting fewer than 200,000 patients in the U.S. Orphan designation qualifies the sponsor of the drug for various development incentives of the Orphan Drug Act (ODA), including seven-year period of U.S. Marketing exclusivity, tax credits for clinical research costs, clinical research trial design assistance, the ability to apply for annual grant funding, and waiver of Prescription Drug User Fee Act (PDUFA) for filing fees.

OCU300 is a re-purposed drug being developed through the FDA's 505(b)(2) pathway for the treatment of oGVHD. According to a post-hoc analysis of OCU300 administered to patients with oGVHD in an exploratory observational study, there was beneficial effect in approximately 90 percent of patients without significant side effects.