FDA Grants Fast Track Status to Aegle Therapeutics for Dystrophic Epidermolysis Bullosa Treatment

Rare Daily Staff

The U.S. Food and Drug Administration has granted Fast Track designation to Aegle Therapeutics’ AGLE-102 for the treatment of patients with dystrophic epidermolysis bullosa, a rare genetic pediatric skin blistering disorder.

Dystrophic epidermolysis bullosa (DEB) is one of the major forms of epidermolysis bullosa, with signs and symptoms varying widely among affected individuals. In mild cases, blistering may primarily affect the hands, feet, knees, and elbows. Severe cases involve widespread blistering that can lead to vision loss, disfigurement, and other serious medical problems. Researchers classify DEB into three major types. Although the types differ in severity, their features overlap significantly. All three types are caused by mutations in the COL7A1 gene. The most severe types of dystrophic epidermolysis bullosa are inherited in an autosomal recessive pattern. A milder form of dystrophic epidermolysis bullosa has an autosomal dominant pattern of inheritance.

Aegle Therapeutics is developing a first in class therapy using extracellular vesicles (EV), including exosomes, secreted by allogeneic bone marrow derived mesenchymal stem cells to treat severe dermatological conditions.

AGLE-102 is an extracellular vesicle (EV) therapy that delivers proteins, genetic material and regenerative healing factors to diseased and damaged tissue.  AGLE-102 will be evaluated in DEB patients in a phase 1/2a trial that will start in 2021.

The Fast Track program is intended to facilitate the development and review of drug candidates that treat serious conditions and fill an unmet medical need. A drug candidate with Fast Track designation is eligible for greater access to the FDA for the purpose of expediting the drug product candidate’s development, review and potential approval.

“We are pleased to have received Fast Track designation for AGLE-102. Aegle’s EV therapy is unique in that it delivers collagen 7 protein, COL7A1 mRNA and regenerative healing factors to potentially address the complex nature of DEB,” said Evangelos Badiavas, chief scientific officer at Aegle Therapeutics. “This designation will expedite the development and regulatory review of AGLE-102 and highlights the importance of providing novel treatments to this patient population.”

Photo: Evangelos Badiavas, chief scientific officer at Aegle Therapeutics