" class="no-js "lang="en-US"> Aurion Biotech Receives Approval from in Japan for New Drug
Saturday, April 20, 2024

Aurion Biotech Receives Approval from Japan’s PMDA for New Drug Application

Aurion Biotech, whose mission is to restore vision to millions of patients with its life-changing regenerative therapies, today announced it has received regulatory approval in Japan from the Pharmaceuticals and Medical Devices Agency (PMDA) for its novel cell therapy, Vyznova™, for the treatment of bullous keratopathy of the cornea. The Company believes this is the first-ever regulatory approval in the world for an allogeneic cell therapy to treat corneal endothelial disease.

“The approval of Vyznova™ in Japan represents many firsts – for millions of patients in need, for regenerative medicine and for Aurion Biotech, including the first-ever approval of a cell therapy to treat corneal endothelial disease,” said Greg Kunst, chief executive officer, Aurion Biotech. “The Aurion team, with deep experience in cell therapy R&D and manufacturing, is leveraging the landmark inventions of Professor Shigergu Kinoshita, MD, PhD and his team, into high-scale clinical cell therapy development – first in Japan, and then throughout the world.”

The breakthrough innovation of this cell therapy is to enable fully differentiated corneal endothelial cells (CECs) to regenerate outside the body. Healthy cells from a donor cornea are cultured in a novel, multi-step, proprietary and patented process that produces off-the-shelf, allogeneic, fully differentiated CECs. Without gene editing, fully differentiated CECs from a single donor can be produced to treat more than 100 recipient eyes. The endothelial cells are injected intracamerally where they re-populate into a healthy mono-layer and start removing fluid from the cornea, thereby decreasing corneal edema.

“Restoring corneal endothelial health to patients by innovating with cell therapy has been my life’s work,” said Professor Shigeru Kinoshita, MD, PhD, of Kyoto Prefecture University of Medicine. “PMDA approval is an essential step in bringing this cell therapy to patients in need in Japan. Vyznova™ cell therapy offers the potential to completely transform the treatment paradigm for corneal endothelial disease, with an ample supply of fully differentiated, allogeneic corneal endothelial cells; a minimally invasive, elegant procedure; and potentially less onerous recovery for patients.”

Epidemiologists estimate that over 4% of adults aged 40+, or approximately 16 million people in the US, EU and Japan, suffer from corneal endothelial diseases such as Fuchs Dystrophy; additional patients suffer endothelial cell damage from ocular surgical trauma. Corneal endothelial cells (CECs) do not regenerate in the eye and decrease in number throughout a person’s lifetime. When disease or injury damages these cells, they are further depleted, and if left untreated, patients may lose vision. Current standards of care, including penetrating keratoplasty (PK) and endothelial keratoplasty (EK), are complex procedures with potentially difficult post-operative recoveries and complications, such as graft detachment, transplant rejection, dislocation, irregular astigmatism, and infection. In addition, these procedures require the use of one donor cornea for each corneal transplant. Yet, demand for donor corneas far exceeds supply: it’s estimated that there is only one donor cornea available for every 70 diseased eyes.

To date, 130 subjects have been treated with Aurion Biotech’s cell therapy procedure in Japan and other OUS studies. In these studies, subjects have experienced clinically meaningful and sustained improvements in key measures of corneal health: visual acuity, central corneal thickness, and endothelial cell density. The company anticipates starting clinical studies evaluating the treatment of patients with corneal endothelial disease with its endothelial cell therapy in the United States later this year.

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