FDA grants Rare Pediatric Disease (RPD) designation to Atsena Therapeutics ATSN-101 for Leber congenital amaurosis

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Atsena Therapeutics announced the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) designation to ATSN-101, the company’s investigational gene therapy being evaluated in an ongoing Phase I/II clinical trial in patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D (LCA1). The FDA previously granted Regenerative Medicine Advanced Therapy (RMAT) designation and orphan drug designation to ATSN-101 for the treatment of LCA1.¹

The FDA’s RPD designation is granted for serious or life-threatening diseases which affect fewer than 200,000 people in the US and in which the serious or life-threatening manifestations primarily affect individuals less than 18 years of age. Furthermore, if a Biologics License Application for ATSN-101 for the treatment of LCA1 is approved by the FDA, Atsena may be eligible to receive a Priority Review Voucher that can be redeemed to receive a priority review for any subsequent marketing application or may be sold or transferred.¹

Patrick Ritschel, MBA, Chief Executive Officer of Atsena Therapeutics shared the signifigance of this designation and the need for treatment for this condition saying, “Rare Pediatric Disease designation is a significant milestone for our LCA1 program as we explore options to advance ATSN-101 into a pivotal clinical trial. The FDA designations that have been granted to ATSN-101 not only emphasize the tremendous need for a treatment for patients with LCA1, but also the potential for our subretinal gene therapy to be a major advance in reversing pediatric blindness.”¹

The company plans to present positive 12-month safety and efficacy data from the company’s ongoing Phase I/II clinical trial of ATSN-101 at the 47th Annual Macula Society Meeting. This meeting is schedule to be held February 7-10, 2024, in Palm Springs, CA.¹

Title: Twelve-month safety and efficacy of ATSN-101 in patients with Leber congenital amaurosis caused by biallelic mutations in GUCY2D (LCA1)

Session: Inherited Retinal Dystrophy I (Treatment Trials)

Date and Time: Wednesday, February 7, 2024, 6:04 pm PST

Presenter: Christine Nichols Kay, MD

Reference:

1. Atsena Therapeutics Receives Rare Pediatric Disease Designation from FDA for ATSN-101 Gene Therapy for GUCY2D-associated Leber Congenital Amaurosis (LCA1). Atsena Therapeutics. Published January 16, 2024. Accessed January 16, 2024. https://atsenatx.com/press-release/atsena-therapeutics-receives-rare-pediatric-disease-designation-from-fda-for-atsn-101-gene-therapy-for-gucy2d-associated-leber-congenital-amaurosis/