FDA Reject Amryt’s EB Therapy

The U.S. Food and Drug Administration said it would not approve Amryt’s application for Oleogel-S10, a treatment for the cutaneous manifestations of dystrophic and junctional epidermolysis bullosa, a rare, genetic skin disorder for which there are no approved treatment options.

The FDA said that it had completed its review of the application and has determined that the application cannot be approved in its present form. The FDA has asked Amryt to submit additional confirmatory evidence of effectiveness for Oleogel-S10 in EB. Amryt said it will discuss with the FDA the nature of the data required to address the agency’s concerns.

EB is a rare group of hereditary disorders of the skin, mucous membranes, and internal epithelial linings characterized by extreme skin fragility and blister development. Patients with severe forms of EB suffer from severe chronic blistering, ulceration, and scarring of the skin, mutilating scarring of the hands and feet, joint contractures, strictures of the esophagus and mucous membranes, a high risk of developing aggressive squamous cell carcinomas, infections, and risk of premature death.

Amryt’s lead development candidate, Oleogel-S10 is a potential treatment for the cutaneous manifestations of junctional and dystrophic epidermolysis bullosa (EB), a rare and distressing genetic skin disorder affecting young children and adults for which there is currently no approved treatment. Filsuvez has been selected as the brand name for Oleogel-S10. The product does not currently have regulatory approval to treat EB.

“EB is a devastating disease with no approved treatment options and the EB community is desperate for a therapy,” said Joe Wiley, CEO of Amryt. “The EASE study was the largest randomized clinical trial ever conducted in this disease and the first phase 3 study to meet its primary endpoint. We are committed to working collaboratively with the FDA to identify the most expeditious pathway towards a potential approval for this important therapy,” said Joe Wiley, CEO of Amryt.”

Photo: Joe Wiley, CEO of Amryt

Author: Rare Daily Staff