Abeona Therapeutics Inc (NASDAQ:ABEO) Receives FDA Orphan Drug Designation
Abeona Therapeutics Inc (NASDAQ:ABEO) has announced receiving an Orphan Drug Designation (ODD) from the U.S. Federal Drug Administration (FDA) for its EB-101 gene therapy targeting dystrophic epidermolysis bullosa (DEB) patients. This also includes recessive dystrophic epidermolysis bullosa (RDEB), a deadly genetic skin disorder characterized by lesions and skin blisters. Abeona specializes in the development of gene therapies for treatment of life-threatening diseases.
In a statement, Abeona Therapeutics Inc (NASDAQ:ABEO) President & CEO, Timothy J. Miller, Ph.D said Abeona’s main agenda is to develop therapies to address the huge unmet demands from patients suffering from dystrophic epidermolysis bullosa. He added that they highly grateful for the new designation and the recognition of EB-101 by FDA as a rare product which will offer much needed therapeutic benefits to patients suffering from dystrophic types of epidermolysis bullosa among them RDEB. EB-101 is the company’s third gene therapy to be given the Orphan Drug Designation by the FDA that, as Miller states, is an important driver to the company’s program as it offers seven years of therapies for the same indications.
Wounds that appear on patients with RDEB, referred to as “butterfly skin” syndrome, can go unhealed for several months or even years as a result of the inability of the skin to remain attached to the dermis below and can spread over a large part of the body. During the Phase 1/2 clinical study, EB-101 was used to treat non-healing chronic wounds on each patient and examined the process of healing at particular time intervals over years. The clinical trial’s endpoint examined the safety as well as evaluating the healing process and compared the results to control untreated wounds. The secondary endpoint of the trial includes collagen C7 as well as the restoration of anchoring fibrils after three and six months of post-treatment.
EB is a class of deadly genetic skin diseases which leads to growth of erosions and blisters on the skin and spreads over the entire body. The most deadly is the epidermolysis bullosa (RDEB), which is known for open and painful wounds, skin blistering, esophageal strictures, joint contractures, corneal abrasions, pseudosyndactyly and a shortened life span. Patients suffering from RDEB have a shortage of type VII collagen (C7) as a results of gene mutations in the gene COL7A1 which encodes for C7 and the main element of anchoring fibrils, which make the connection between the dermis and the epidermis.
Abeona Therapeutics Inc (NASDAQ:ABEO) closed Thursday’s session at $4.75 losing 4.04%.
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About the author: Monica has an undergraduate degree in Accounting and an MBA she earned – with Honors. She has six years of experience in the financial markets and has been an analyst for the past two years.