FDA grants fast track designation to X-linked retinoschisis treatment
Key takeaways:
- ATSN-201 is being evaluated for the treatment of X-linked retinoschisis.
- The gene therapy previously received orphan drug and rare pediatric disease designations.
Editor’s note: This is a developing news story. Please check back soon for updates.
The FDA granted fast track designation to ATSN-201 for the treatment of X-linked retinoschisis, according to a press release from Atsena Therapeutics.
“The diseases for which Atsena is developing therapies currently have no approved treatments,” Kenji Fujita, MD, chief medical officer of Atsena, told Healio. “By replacing the defective gene with a healthy copy, Atsena’s approach addresses the root of the pathology.”
The therapy is designed to yield therapeutic levels of gene expression in the photoreceptors of the central retina while reducing the surgical risks for foveal detachment through the use of the company’s AAV.SPR spreading capsid. It is being investigated in the phase 1/2 LIGHTHOUSE study in male patients aged 6 years and older with X-linked retinoschisis (XLRS) caused by mutations in the RS1 gene.
“The AAV technology allows for safer administration of treatment,” Fujita told Healio. “Because the bleb doesn’t have to be injected at the fovea, the surgeon can select more peripheral sites that are unaffected by schisis. Additionally, the surgeon can administer multiple small blebs rather than a single large one.”
The fast track designation is granted to treatments that may address unmet medical needs in serious or life-threatening diseases and may provide Atsena with “more frequent interactions with the FDA throughout drug development,” the release said. Additionally, if relevant criteria are met, treatments on the fast track program may receive priority review.
As Healio reported, the gene therapy previously received orphan drug designation and rare pediatric disease designation for XLRS. There are no approved treatments for the disease.
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