Abeona Faces Clinical Hold from the FDA Ahead of Epidermolysis Bullosa Trial

According to a story from globenewswire.com, the gene and cell therapy company Abeona Therapeutics Inc. recently announced that has received a letter from the US Food and Drug Administration (FDA) announcing a clinical hold. A clinical hold is an order to halt further trial activities, usually until certain conditions are met. In this case, the agency is withholding approval for the company’s phase 3 trial until data concerning transportation stability of the investigational drug being tested is submitted. This trial will test EB-101, an experimental cell therapy being developed as a treatment for recessive dystrophic epidermolysis bullosa.

About Epidermolysis Bullosa

Epidermolysis bullosa is a group of genetic conditions which are most characterized by unusual blistering of the mucous membranes and skin. These painful blisters can occur with only minor friction or abrasion. The disease can be fatal in severe cases. These disorders are all caused by genetic mutations; the various subtypes of epidermolysis bullosa are classified by which genes are affected. Recessive dystrophic epidermolysis bullosa is caused by mutations affecting the COL7A1 gene and is characterized by debilitating oral lesions. Painful mucosal and skin blisters are the defining symptoms, but other complications may include esophageal narrowing and a vastly increased risk of skin cancer. There is no cure for the condition; treatment primarily focuses on symptom management, such as controlling pain, cleaning wounds, prevention or treatment of complications, nutritional support, and controlling infections. Males and females are affected at equal rates. The disease can seriously shorten lifespan, and in severe cases, patients do not survive beyond infancy. There is a dire need for more effective treatment approaches for epidermolysis bullosa. To learn more about these disorders, click here.

The company plans to address the FDA’s request as quickly as possible and still anticipates Chemical, Manufacturing, and Controls (CMC) clearance for the trial by Q4 of this year.

About EB-101

EB-101 is described as a gene-corrected cell therapy that has entered the advanced stages of development and could represent a major break through for the treatment of recessive dystophic epidermolysis bullosa. The therapy utilizes gene transfer to implant corrected COL7A1 genes into a patient’s skin cells to facilitate normal wound healing. The investigational therapy has earned Orphan Drug status in the US and EU as well as Rare Pediatric designation, Breakthrough Therapy designation, and Regenerative Medicine Advanced Therapy designation.


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