Ocugen wins orphan drug status for ocular gene therapy

The FDA has given Ocugen orphan drug status for a gene therapy designed to treat NR2E3 mutation-associated retinal degenerative disease.

The Malvern, Penn.-based company’s OCU400 product includes an adeno-associated virum serotype 5 capsid that contains the gene for human nuclear hormone receptor NR2E3.

In a mouse model of NR2E3 mutation, delivery of NR2E3 to retinal cells reversed disease progression, according to Ocugen. The company plans to kick off a Phase I/IIa clinical study of OCU400 by 2020.

“We are very pleased to receive our first orphan drug designation for a gene therapy product from the FDA,” co-founder & CEO Shankar Musunuri said in prepared remarks. “Unlike single-gene replacement approaches, which have shown great promise in rare retinal diseases despite being highly specific for a single condition, we believe OCU400 represents a powerful and broad means of treating a variety of genetically diverse IRDs with a single product.

Our initial strategy is to develop OCU400 as a gene augmentation therapy for patients with IRDs caused by mutations in the NR2E3 gene. Orphan designation for this initial indication is not only a major milestone for our Modifier Gene Therapy Platform, it also marks the third product in Ocugen’s clinical pipeline to be granted ODD,” Musunuri added.

Ocugen has two other drug candidates that have been granted orphan drug status by the FDA: OCU100 for retinitis pigmentosa and OCU300 for ocular graft versus host disease.

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