Summation
- Breakthrough Therapy Designation is a process designed to expedite the development and review of drugs that are intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s).
Aurion Biotech, whose mission is to restore vision to millions of patients with life-changing regenerative therapies, today announced that the U.S. Food and Drug Administration (FDA) has granted both Breakthrough Therapy Designation (BTD) and Regenerative Medicine Advanced Therapy (RMAT) Designation for AURN001, the Company’s allogeneic cell therapy candidate for the treatment of corneal edema secondary to corneal endothelial disease.
Breakthrough Therapy Designation is a process designed to expedite the development and review of drugs that are intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s).
RMAT is part of the 21st Century Cures Act1. An investigational drug is eligible for RMAT designation if: it meets the definition of regenerative medicine therapy; is intended to treat, modify, reverse, or cure a serious condition; and preliminary clinical evidence indicates that the regenerative medicine therapy has the potential to address unmet medical needs for such conditions.
Sponsor companies receiving BTD and RMAT Designation can benefit from increased interactions with the FDA with the goal of expediting drug development. The dual designations granted to Aurion Biotech are based on the criteria stated above, the unmet medical need among patients with corneal endothelial disease, and FDA’s review of clinical data from the AURN001 program, which includes multiple clinical trials with subjects having completed at least 12 months of follow-up.
“Aurion Biotech is honored to receive the Breakthrough Therapy Designation and Regenerative Medicine Advanced Therapy Designation for AURN001,” said Sterling Chung, Vice President of Regulatory Affairs and Quality at Aurion Biotech. “These designations underscore the importance of developing a potential solution for millions of patients around the world who suffer from corneal endothelial diseases. We look forward to working closely with the FDA to expedite the development of our cell therapy.”
This news comes on the heels of Aurion’s announcement that it has completed enrollment and dosing of its Phase 1 / 2 CLARA trial in the U.S. and Canada2. The CLARA clinical trial is a prospective, multi-center, randomized, double-masked, parallel-arm cell dose-ranging study in subjects with corneal edema secondary to corneal endothelial dysfunction. The study will evaluate three different doses of neltependocel used in combination with Y-27632. Ninety-seven subjects have been randomized at US & Canadian sites. The CLARA trial is designed to assess the safety, tolerability, and efficacy of AURN001 for the treatment of corneal edema secondary to corneal endothelial dysfunction. The primary endpoint is the percentage of subjects who gain three (3) lines of vision at six (6) months.
The company has already received regulatory approval in Japan.
“The BTD and RMAT designations are important milestones for people living with corneal endothelial disease who need better, less burdensome, more accessible treatment options,” said Greg Kunst, chief executive officer of Aurion. “I thank the Aurion team for their hard work and dedication, and I’m grateful for the essential contributions of clinical trial patients and their ophthalmologists – all of whom have helped to make today’s news a reality.”
About Corneal Endothelial Dysfunction: Corneal edema secondary to endothelial dysfunction is a sight-threatening and debilitating condition affecting millions of people throughout the world. When corneal endothelial cells die or degrade, they do not regenerate. If left untreated, corneal endothelial cell loss can cause corneal edema (swelling) and loss of vision. Although corneal transplants are effective, there are disadvantages with these procedures – PKP/DMEK/DSAEK – including limited donor organ supply. Transplants require a supply of donor corneas in a 1:1 ratio (one healthy donor cornea to treat each diseased one), yet it’s estimated that there is only one donor cornea available for every 70 diseased eyes. In addition, post-operative recovery for corneal transplant patients requires that they lie flat on their backs for up to three days, in order for the transplant to adhere to the corneal stroma.