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


Intermediate/Posterior non-infectious Uveitis

Ophthotech is currently planning a small, open-label Phase 2a clinical trial of Zimura monotherapy for the treatment of non-infectious intermediate and posterior uveitis, a rare inflammatory disease of the back of the eye. The FDA has recognized non-infections intermediate and posterior uveitis as an orphan disease, with several treatments either approved or in development having received orphan product designation from the FDA. This trial is scheduled to initiate during 2018.


Patient Resources

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.