Ophthalmic Orphan Programs

Science driven, retina focused company with multiple programs concentrated in orphan indications

Stargardt Disease

Ophthotech’s orphan ophthalmic disease strategy will be led by a randomized, controlled clinical trial assessing the safety and efficacy of Zimura® (avacincaptad pegol), our C5 complement inhibitor, for Stargardt disease, a devastating inherited retinal orphan disease causing vision loss during childhood or adolescence for which patients have no FDA or EMA approved treatment. This trial is on-going.

Ophthotech has engaged Foundation Fighting Blindness (FFB), a highly-distinguished organization recognized for its scientific commitment to orphan inherited retinal degenerative diseases with an established network of scientists and a robust patient registry, to provide the Company with information from its publicly available ProgStar study, the largest natural history study on Stargardt disease to date, which Ophthotech was used in the design of its clinical trial of Zimura for Stargardt disease.

Stargardt disease is a devastating inherited retinal orphan disease that causes vision loss during childhood/adolescence with no FDA or EMA approved treatment option. There is a high unmet medical need for a treatment option. The estimated prevalence in the US is approximately 32,000 to 41,000 patients.1

To find out more about the symptoms and signs of AMD, please refer to these sites. Ophthotech is not responsible for the information provided by these organizations.

Clinical Trials




  1. Blacharski PA. Fundus flavimaculatus. In: Newsome DA ed. Retinal Dystrophies and Degenerations. New York: Raven Press; 1988:135–159.