Pharma Focus America

Aurion Biotech Has Been Granted Breakthrough Therapy Designation and Regenerative Medicine Advanced Therapy Designation for Its Drug Candidate AURN001

Thursday, June 20, 2024

Aurion Biotech has announced that the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation (BTD) and Regenerative Medicine Advanced Therapy (RMAT) Designation to AURN001. This therapy is designed to treat corneal edema resulting from corneal endothelial disease.

Breakthrough Therapy Designation aims to expedite the development and review of drugs for serious conditions where preliminary clinical evidence suggests substantial improvement over existing therapies. RMAT Designation, part of the 21st Century Cures Act, applies to regenerative medicine therapies addressing unmet medical needs.

The designations recognize AURN001's potential based on clinical data from multiple trials involving subjects followed for at least 12 months.

Sterling Chung, Vice President of Regulatory Affairs and Quality at Aurion Biotech, emphasized the significance of these designations for advancing treatments for corneal endothelial diseases. Aurion looks forward to collaborating closely with the FDA to accelerate the therapy's development.

This announcement follows completion of patient enrollment and dosing in Aurion's Phase 1/2 CLARA trial in the U.S. and Canada. The study evaluates different doses of neltependocel in combination with Y-27632 for treating corneal edema due to endothelial dysfunction. The primary goal is to assess safety, tolerability, and efficacy, measuring improvement in vision at six months.

Aurion has also secured regulatory approval for AURN001 in Japan.

Aurion Biotech, underscored the importance of these designations in advancing treatment options for corneal endothelial disease, thanking the Aurion team, clinical trial participants, and their ophthalmologists for their contributions.



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