ATSN-101, a gene therapy for GUCY2D-associated Leber congenital amaurosis, has demonstrated clinically meaningful improvements in vision at the highest dose with no drug-related serious adverse events 6 months post-treatment in ongoing Phase I/II clinical trial.
Atsena Therapeutics announced the US Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to ATSN-101, the company’s lead investigational gene therapy for patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D (LCA1).
According to the company, the RMAT designation was granted based on positive 6-month efficacy data from the company’s ongoing Phase I/II clinical trial of ATSN-101.
“Receiving RMAT designation from the FDA for ATSN-101 marks a significant regulatory milestone for Atsena, validating the potential of our subretinal gene therapy to improve vision and make a meaningful difference in the lives of patients with LCA1,” Patrick Ritschel, MBA, CEO of Atsena Therapeutics, said in a news release. “As we continue to explore options to advance ATSN-101 into a pivotal clinical trial, we look forward to reporting 12-month data from our ongoing Phase I/II trial by the end of this year.”
“There are no approved treatments for LCA1, an inherited retinal disease that results in early and profound vision impairment or blindness,” said Jason Menzo, CEO of Foundation Fighting Blindness. “RMAT designation is encouraging recognition of the potential of ATSN-101 to be an important treatment and provides hope to children and adults affected by LCA1.”
Established under the 21st Century Cures Act, RMAT designation is a dedicated program designed to expedite the drug development and review processes for promising pipeline products, including gene therapies. A regenerative medicine therapy is eligible for RMAT designation if it is intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition, and preliminary clinical evidence indicates that the drug or therapy has the potential to address unmet medical needs for that disease or condition.1
According to the company’s news release, RMAT designation provides sponsors with intensive FDA guidance on efficient drug development, including the ability to discuss surrogate or intermediate endpoints, potential ways to support accelerated approval and satisfy post-approval requirements, potential priority review of the biologics license application (BLA), and other opportunities to expedite development and review.
Atsena noted in its news release has also received orphan drug designation from the FDA for ATSN-101 for the treatment of LCA1.
LCA1 is a monogenic eye disease that disrupts the function of the retina. It is caused by mutations in the GUCY2D gene and results in early and severe vision impairment or blindness. GUCY2D-LCA is one of the most common forms of LCA, affecting roughly 20 percent of patients who live with this group of inherited retinal diseases. There are currently no approved treatments for LCA1.1