2023-04-27 08:52:21 ET
- The U.S. FDA granted orphan drug designation (ODD) to Ocugen's ( NASDAQ: OCGN ) gene therapy OCU410ST (AAV5- hRORA) to treat certain vision disorders.
- The ODD was given for treating ABCA4-associated retinopathies, including Stargardt, Retinitis Pigmentosa 19 (RP19), and Cone-rod dystrophy 3 (CORD3) diseases.
- OCU410ST uses an AAV delivery platform for the retinal delivery of the RORA (RAR Related Orphan Receptor A) gene and represents the company's modifier gene therapy approach, according to Ocugen.
- "There are approximately 44,000 patients in the U.S. living with ABCA4-associated retinal diseases for whom no treatment options exist," said Shankar Musunuri, co-founder, chairman and CEO, Ocugen.
- The company plans to submit an application in Q2 to start a phase 1/2 trial.
- The FDA grants orphan drug status to therapies which treat or prevent rare diseases that affect fewer than 200K people in the U.S. The designation provides certain incentives, including seven years of market exclusivity, if approved.
- OCGN +1.11% to $0.73 premarket April 27
For further details see:
Ocugen's gene therapy for vision disorder gets FDA orphan drug tag