FDA grants Rare Pediatric Disease designation for ATSN-201 for the treatment of XLRS
The LIGHTHOUSE study a Phase I/II, open-label, dose-escalation and dose-expansion clinical trial is currently investigating the safety and tolerability of ASTN-201 for XLRS.
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The US Food and Drug Administration (FDA) has granted Rare Pediatric Disease designation (RPD) for ATSN-201 for the treatment of X-linked retinoschisis (XLRS). ATSN-201, developed by Atsena Therapeutics, is a gene therapy product candidate that uses AAV.SPR, which is the company’s novel spreading capsid. This candidate is used to achieve therapeutic levels of gene expression in photoreceptors of the central retina while avoiding the surgical risks of foveal detachment.1
The FDA grants Rare Pediatric Disease designation to therapeutics intended to treat serious or life-threatening rare diseases that primarily affect individuals under the age of 18.
Patrick Ritschel, Chief Executive Officer of Atsena Therapeutics shared how this designation empowers the company to meet the need of patients with XLRS. In the press release1, he said, “We are pleased to receive the FDA’s Rare Pediatric Disease designation for ATSN-201, which also marks the second RPD designation granted to Atsena this year. Having both of our clinical-stage, ocular gene therapies receive this designation underscores the potential of our technology to address significant unmet needs for patients with inherited retinal diseases. We are committed to advancing ATSN-201 in clinical trials and offering hope to patients and families affected by XLRS.”
As of publication, there are no approved treatments for XLRS on the market, meaning there is unmet need for patients diagnosed with XLRS. Typically, patients with this condition are diagnosed in early childhood, with there being a larger number of males affected. Approximately 30,000 males in the US and EU are affected by XLRS.1
The LIGHTHOUSE study (NCT05878860), a Phase I/II, open-label, dose-escalation and dose-expansion clinical trial is currently investigating the safety and tolerability of ASTN-201. The trial participants are male patients ages 6 and older with a clinical diagnosis of XLRS caused by mutations in the RS1 gene.1
