Atsena Therapeutics announced that the FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to ATSN-101, the company's primary investigational gene therapy designed for individuals with Leber congenital amaurosis caused by biallelic mutations in GUCY2D (LCA1).
The RMAT designation is in recognition of favorable 6-month efficacy data obtained from Atsena Therapeutics' ongoing phase 1/2 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, Chief Executive Officer of Atsena Therapeutics, said in a company 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 1/2 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,” Jason Menzo, Chief Executive Officer of Foundation Fighting Blindness, said in a company news release. “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 through the 21st Century Cures Act, the RMAT designation operates as a specialized program aimed at streamlining the development and review processes for promising products in the pipeline, specifically including gene therapies. For a regenerative medicine therapy to qualify for RMAT designation, it must have the intent to treat, modify, reverse, or cure a serious or life-threatening disease or condition. Additionally, preliminary clinical evidence is required to indicate that the therapy holds the potential to address unmet medical needs for that particular disease or condition.
RMAT designation offers sponsors comprehensive FDA guidance on efficient drug development. This includes discussions on surrogate or intermediate endpoints, potential methods to support accelerated approval, meeting post-approval requirements, the prospect of priority review for the biologics license application (BLA), and other avenues to expedite the development and review processes.