In the United States, Fast Track designation is granted to drugs designed to treat rare and/or serious conditions and that fill an unmet medical need. The process aims to get drugs into the hands of patients more efficiently. Fast Track designation also comes with more frequent written communication and meetings with the FDA, Rolling Review, and eligibility for Priority Review and Accelerated Approval. According to a news release from biotechnology company Endogena Therapeutics Inc. (“Endogena”), the company’s therapy EA-2353 recently earned Fast Track designation for patients with retinitis pigmentosa (RP).
EA-2353 is a novel, small-molecule treatment. It activates endogenous retinal stem and progenitor cells. These cells, as they become differentiated, may serve to protect visual function and stength. EA-2353 is administered intravitreally. At the moment, researchers are evaluating the safety, efficacy, of tolerability of this therapy within a Phase 1/2a study. Within the study, participants will receive one unilateral intravitreal injection every week for a one month period.
Outside of Fast Track designation, EA-2353 also earned Orphan Drug designation two years ago.
About Retinitis Pigmentosa (RP)
Retinitis pigmentosa refers to a collective of rare inherited eye diseases which affect the retina, a thin and light-sensitive piece of tissue at the back of the eye. In RP, rods and cones (cells in the eye) break down and degenerate. This causes progressive vision loss. Somewhere between 60-100 different genes have been implicated in retinitis pigmentosa development. Because there are multiple forms of RP, there are also different patterns of inheritance: autosomal recessive, autosomal dominant, and X-linked.
Symptoms vary based on whether cones or rods are affected first. In most cases, rods are affected first. Overall, symptoms related to RP can, but do not always, include:
- Decreased peripheral and central vision
- Sensitivity to bright light
- Night blindness
- Changes in visual acuity and color perception
- Reduced ability to perceive details
Many people with RP are legally blind by 40 years old.
