FDA grants orphan drug designation for ATSN-201 for X-linked retinoschisis
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ATSN-201 received orphan drug designation from the FDA for the treatment of X-linked retinoschisis, according to a press release from Atsena Therapeutics.
“There are currently no approved treatments for XLRS patients of any age,” Patrick Ritschel, CEO of Atsena Therapeutics, told Healio. “Our program is evaluating the safety and efficacy of ATSN-201 to treat the underlying genetic cause of XLRS in both adults and children with a one-time subretinal injection. This designation, along with the rare pediatric disease designation received earlier this year, gives us confidence in the value of this program and the potential benefit that it may be able to bring to individuals impacted by this rare disease.”
The gene therapy uses the company’s AAV.SPR spreading capsid to achieve gene expression in the photoreceptors of the central retina without increased risk for foveal detachment.
In addition to benefits such as tax credits toward the cost of clinical trials if ATSN-201 is approved and prescription drug user fee waivers, the designation provides 7 years of market exclusivity for ATSN-201 for the treatment of X-linked retinoschisis (XLRS).
“We continue enrolling our phase 1/2 trial and expect to begin including pediatric participants early next year,” Ritschel said. “With this designation, we look forward to having collaborative discussions with FDA around the design of a phase 3 clinical trial to expedite our path toward approval of ATSN-201 for XLRS.”
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