According to a story from Benzinga, the drug company Abeona Therapeutics, Inc. has recently announced plans to move forward with its phase 3 clinical trial following the lifting of the study’s clinical hold by the US Food and Drug Administration (FDA). This trial will test the company’s investigational therapy candidate EB-101 as a treatment for the recessive dystrophic form of epidermolysis bullosa, a rare disease that affects the skin.
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. Painful mucosal and skin blisters are the defining symptoms, but other complications may include esophageal narrowing and a vastly increased risk of skin cancer. The recessive dystrophic form is very severe and causes debilitating and painful oral lesions. 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 hold on the trial was lifted after Abeona complied with the FDA’s request to submit more data in regards to the stability of the medication during transportation to clinical trial sites. The company’s current time table has the phase 3 trial beginning in the first quarter of 2020. Abeona is excited about the future of EB-101 as the results of previous trials have suggested that the experimental drug could have a meaningful positive impact on large wound healing for patients with the recessive dystrophic variant of epidermolysis bullosa.
EB-101 is classified as a gene-corrected cell therapy in which COL7A1 genes are delivered to a patient’s skin cells in the lab setting. These altered cells are then transplanted back to the patient’s body, which should enable normalized expression of Type VII collagen. This in turn allows for patients to experience wound healing.