The FDA Grants Ocugen Inc. (NASDAQ:OCGN) the Fourth Orphan Drug Designation (ODD) for OCU400 Gene Therapy

Ocugen Inc. (NASDAQ:OCGN) is one of the forces to reckon in the development of innovative eye therapeutics. The company’s product lines aim to therapy needs of patients with underserved diseases including age-related macular degeneration. The latest is OCU400, a treatment for PDE6B gene mutation-associated retinal diseases and which has received the fourth Orphan Drug Designation (ODD) from the FDA.

OCU400 has the potential of restoring retinal integrity, which allows the proper functioning of the eyes across a range of genetically diverse inherited retinal diseases.

Ocugen’s Modifier Gene Therapy Platform

PDE6B mutation causes retinitis Pigmentosa (RP) inherited retinal dystrophy. By midlife, the victim already has blindness followed by progressive loss of photoreceptors. Statistics indicate that a single mutation in the PDE6B gene can cause the inability to see in low light, commonly referred to as congenital stationary night blindness.

However, the breakthrough platform has a unique approach to addressing multiple retinal diseases with just one drug. This could serve as a reliever, which could also lay a basis for a smoother regulatory pathway in the recovery of development costs over multiple therapeutic indications. The ODD for PDE6B gene mutation will offer such significant support to the breakthrough modifier gene therapy platform, according to Carl D. Regillo, a member of Ocugen’s Retina Scientific Advisory Board.

‘’… I have been on the cutting-edge of many new ophthalmology treatments. I am very encouraged by the potential for OCU400 given the uniqueness of Ocugen’s Modifier Gene Therapy Platform…’’, the principal investigator of several clinical trials commented.

More than 150 Gene Mutations Are Affecting Over 1.5 Million Individuals Worldwide   

The co-Founder and CEO of Ocugen are thrilled about the company’s latest accomplishment. Dr. Shankar Musunuri says that such milestones keep their goals enhanced. The most unfortunate thing is that there are more 1.5Milion globally suffering from over 150 gene mutations, yet there are no approved treatments to stop or slow their progression.

A majority of them rely on traditional therapy or gene editing approaches, which requires an additional 150 products to rescue them from vision loss. All in all, Ocugen remains dedicated to developing transformative therapies.