US FDA granted ODD for Abeona’s EB-101 gene therapy program

US FDA has granted Orphan Drug Designation (ODD) for Abeona’s EB-101 gene therapy program for patients with dystrophic epidermolysis bullosa (DEB), including recessive dystrophic epidermolysis  bullosa (RDEB).

Abeona Therapeutics President & CEO,  Timothy J. Miller said that Abeona is committed to advancing innovative gene therapies that address the unmet needs of patients suffering with dystrophic epidermolysis bullosa, a devastating rare skin disease. They are grateful that the FDA has recognized EB-101 as a rare disease product that may offer a significant therapeutic benefit for patients with dystrophic forms of epidermolysis bullosa, including RDEB. EB-101 is Abeona’s third gene therapy program to be granted Orphan Drug Designation by the FDA, an important value driver for the program as it provides seven years of market exclusivity from similar medicines for similar indications.

Typically, wounds on patients with RDEB, also known as “butterfly skin” syndrome, can remain unhealed for months to years due to the inability of the skin to stay attached to the underlying dermis and can cover a large percentage of the body.  In the Phase 1/2 clinical trial, EB-101 was administered to non-healing chronic wounds on each subject and assessed for wound healing at predefined time points over years. The primary endpoints of the clinical trial assess safety and evaluate wound healing after EB-101 administration compared to control untreated wounds. Secondary endpoints include expression of collagen C7 and restoration of anchoring fibrils at three and six months post-administration.

Clinical data were recently presented at the Society of Investigative Dermatology (SID) conference by Stanford collaborators, and demonstrated that EB-101 treated wounds were significantly healed >50% for more than 2 years post-administration. The data showed:

Wound healing, defined as >50% closure after EB-101 administration, was observed in:

–100% (36/36 treated wounds, n=6 subjects) at 3 months,

–89% (32/36 treated wounds, n=6 subjects) at 6 months,

–83% (20/24 treated wounds, n=4 subjects) at 12 months,

–88% (21/24 treated wounds, n=4 subjects) at 24 months,

–100% (6/6 treated wounds, n=1 subject) at 36 months post-administration.

Collagen VII (C7) expression: C7 and morphologically normal NC2 reactive anchoring fibrils – the “zipper” that holds skin onto the underlying tissue and the primary deficit in RDEB patients – were observed in EB-101 treated wounds up to two years post administration.