The U.S. Food and Drug Administration (FDA) recently granted the Rare Pediatric Disease Designation (RPDD) to OCU410ST, a drug developed by Malvern-based Ocugen, a biotechnology company specializing in gene therapies for blindness diseases.
The RPDD is for serious and life-threatening diseases that primarily affect people ages 18 years old and younger and that affect fewer than 200,000 people in the United States.
OCU410ST is a treatment for ABCA4-associated retinopathies including Stargardt disease, retinitis pigmentosa 19, and cone-rod dystrophy 3. It utilizes a modifier gene therapy approach, which is based on Nuclear Hormone Receptor (NHR) RORA that regulates pathophysiological pathways linked to Stargardt disease.
Stargardt disease is the most common form of inherited macular degeneration. It causes retinal degeneration and vision loss, usually with some peripheral vision preserved. It usually develops during childhood or adolescence. Approximately 100,000 people in the United States and Europe are affected.
“This latest designation for OCU410ST reaffirms the urgency of providing a therapeutic option to Stargardt patients who have no FDA-approved treatment available,” Dr. Shankar Musunuri, Ocugen chairman, CEO, and co-founder, said. “This inherited retinal disease presents itself most often in childhood—making Stargardt disease a diagnosis that not only affects the patient but impacts the entire family.”