Atsena Therapeutics announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for ATSN-201 for the treatment of X-linked retinoschisis (XLRS).
ATSN-201, a best-in-class gene therapy product candidate, leverages AAV.SPR, the company’s novel spreading capsid, to achieve therapeutic levels of gene expression in photoreceptors of the central retina while avoiding the surgical risks of foveal detachment.
“We are honored to receive the FDA’s Orphan Drug Designation for ATSN-201, which was also recently granted Rare Pediatric Disease designation. These designations mark a significant inflection point for the potential of this ocular gene therapy in an inherited retinal disease that currently has no available treatments,” said Patrick Ritschel, CEO of Atsena Therapeutics. “The Atsena team is passionate and committed to our ongoing work on the XLRS program. We look forward to bringing hope to patients affected by this rare disease and are confident these designations will expedite our path forward.”
The FDA grants Orphan Drug Designation to drugs and biologics that are intended for safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the U.S. Orphan Drug Designation provides certain incentives, such as tax credits toward the cost of clinical trials upon approval and prescription drug user fee waivers. If a product receives Orphan Drug Status from the FDA, that product is entitled to seven years of market exclusivity for the disease in which it has Orphan Drug designation, which is independent from intellectual property protection.
Currently, there are no approved treatments for XLRS, which is typically diagnosed in early childhood and affects approximately 30,000 males in the U.S. and EU.
The safety and tolerability of ATSN-201 is currently being evaluated in the LIGHTHOUSE study, a Phase I/II, open-label, dose-escalation and dose-expansion clinical trial in male patients ages six and older with a clinical diagnosis of XLRS caused by mutations in the RS1 gene. Enrollment for this study is ongoing. For more information, visit ClinicalTrials.gov (Identifier: NCT05878860).