Skip to content

Update on Abeona's EB-101 cell therapy development for RDEB in the US

The decision from the U.S. Food and Drug Administration (FDA) could be as early as mid-2024, provided the FDA grants the submission Priority Review Status. Typically, the decision-making process takes 10 months after the submission of approval documents, but with Priority Review Status, it would only take 6 months. The FDA has already designated EB-101 as an Advanced Therapy of Regenerative Medicine, a groundbreaking therapy, and an orphan drug for rare diseases and rare pediatric diseases.

What is EB-101?

EB-101 could potentially become the first individualised cell therapy for RDEB patients. It has the potential to offer years of wound healing and pain relief after a single application. However, this therapy has not yet been approved.


How does EB-101 work?

EB-101 works by introducing a functional (healthy) version of the collagen 7 gene into the epidermal cells of the affected person. Consequently, these cells can produce a functional collagen 7 protein, ensuring the cohesion of the skin layers.


How is EB-101 administered?

The functional gene is delivered using a modified virus, which is used to introduce the gene into the cells. The gene-corrected cells are then grown in a laboratory to generate corrected skin, which is subsequently surgically transplanted onto a skin wound. It is expected that the skin graft will produce the collagen 7 protein, promoting wound healing.


What is the basis for potential approval?

The documents submitted to the FDA as a Biological License Application (BLA) are primarily based on data from a phase 3 clinical trial known as VIITAL (NCT04227106). This pivotal trial involved 11 people with RDEB in the US, aged 6 to 40 years, who had wounds that had been open for at least six months. You can find more information in the news article on the EB Resnet website from November 2022.

Back to main navigation