Experimental Stargardt Disease Treatment Earns Orphan Drug and Rare Pediatric Disease Designations

According to a story from PR Newswire, the biopharmaceutical company reVision Therapeutics, Inc. has just announced that its experimental therapy REV-0100 has received both Rare Pediatric Disease designation and Orphan Drug designation from the US Food and Drug Administration (FDA). This investigational treatment is being developed as a treatment for Stargardt disease, a rare illness that can cause blindness.

About Stargardt Disease

Stargardt disease is an inherited disease of the retina, the inner layer of the eye that is sensitive to light. The illness causes macular degeneration, which can begin at different times in the patient’s life. There are different types of Stargardt disease which vary depending on which gene is mutated; in type 1, it is caused by alternations to the ABCA4 gene. In the most severe cases, degeneration can begin in childhood or adolescence, and eventually lead to progressive vision loss. The center of the visual field is most heavily affected; symptoms include blurred vision, impaired color vision, wavy vision, blind spots, loss of depth perception, and poor night vision. Symptoms cannot be corrected with glasses. The pace of vision loss and the symptoms experienced vary from person to person. While there are some lifestyle changes that may slow the progression of Stargardt disease, there is currently no available treatment. Most patients will eventually become legally blind. To learn more about Stargardt disease, click here.

About Orphan Drug Designation

Orphan Drug designation is generally reserved for treatments that are being developed in order to treat rare diseases, which are defined as any illness that affects less than 200,000 people in the US. Such treatments must show potential advantages in efficacy or safety over currently available treatments; alternatively, the therapy must have the potential to address a currently unmet medical need. This designation confers several benefits to the recipient company, such as tax breaks, the waiving of certain fees, and a seven year period of market exclusivity if the FDA approves the drug.

About Rare Pediatric Disease Designation

Rare Pediatric Disease designation is for therapies intended to treat rare diseases that primarily impact people that are eighteen years or younger in age. As in Orphan Drug designation, the treatment must either fulfill an unmet need or have potential advantages when compared to current treatments. The recipient company receives an FDA Priority Review voucher than can be used on a future approval application; alternatively, the voucher can be sold to another company. 

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