RARE Daily

FDA Grants Fast Track Designation to Amryt Pharma for EB Therapy

October 2, 2019

The U.S. Food and Drug Administration granted Amryt Pharma fast track designation for AP101, the company’s experimental treatment for the rare genetic skin disease epidermolysis bullosa.

Photo: Joe Wiley, CEO of Amryt

The fast track designation is designed to facilitate the development and expedite the review of drugs that treat serious conditions, potentially accelerating patient access to new therapies.

Epidermolysis bullosa (EB) can cause skin to blister and tear from the slightest friction or trauma and can, in some cases, cause blistering and erosion of the epithelial lining of internal organs. EB is chronic, potentially disfiguring and in some cases fatal. There are currently no approved treatments.

AP101 is a topical gene therapy that is designed to deliver a functional copy of the collagen gene directly to the skin. It is currently being evaluated in a phase 3 clinical trial, the largest ever global phase 3 study for EB.

“Receiving a fast-track designation from the FDA represents another significant step in the progress of our lead development asset – AP101 – as a potential treatment for the wound related complications of EB, a rare and life limiting condition,” said Joe Wiley, CEO of Amryt. “This designation, alongside the other significant efforts of our team globally, drives our continued progress and brings us closer to realizing our vision of becoming a global leader in rare and orphan diseases.”

Author: Rare Daily Staff

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