Aldeyra Therapeutics (NASDAQ:ALDX) received an orphan drug designation by the US Food and Drug Administration for ADX-102 in treating the rare disease Sjögren-Larsson Syndrome.
As quoted in the press release:
“The skin disease associated with SLS is physically and emotionally debilitating, leading to considerable social stigma and requiring significant daily care and medical resources,” said Todd C. Brady, M.D., Ph.D., President and CEO of Aldeyra. “Receipt of orphan drug designation by the FDA is great step forward for both Aldeyra and patients suffering from this rare and difficult-to-treat disease.”
SLS is caused by genetic mutations that lead to high levels of toxic aldehydes. By sequestering and facilitating the degradation of aldehydes, ADX-102 represents a novel, mechanistically directed potential therapy for SLS. In August of 2016, Aldeyra announced that ADX-102 improved ichthyosis in SLS patients in a randomized, double-blind, vehicle-controlled Phase 2 clinical trial in which ADX-102 was administered to skin for two months. Improvement from baseline was clinically and statistically significant, and the magnitude of improvement was statistically superior to that of vehicle treatment. A global, double-blind, pivotal Phase 3 study is expected to begin later this year. Future clinical trials of ADX-102 or other novel aldehyde traps may involve oral administration as a potential treatment for the neurological aspects of the disease.
The FDA Office of Orphan Products Development designates orphan status to drugs intended to treat, diagnose, or prevent rare diseases that affect fewer than 200,000 people in the United States. Receiving Orphan Drug Designation provides Aldeyra with multiple benefits, including waiver of the Prescription Drug User Fee, post-approval marketing exclusivity for seven years, research tax credits, and assistance during the marketing registration process.