FDA grants Amryt Pediatric Rare Disease Designation for Experimental Drug to Treat Epidermolysis Bullosa

Rare Daily Staff

The U.S. Food and Drug Administration granted Amryt Pharma a Rare Pediatric Disease designation for AP101, the company’s experimental treatment for the rare genetic skin disease 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 currently in a phase 3 clinical trial, the largest ever global phase 3 study for EB. An interim efficacy data readout is due later this year and the top-line data readout is expected in the second quarter of 2019.

The FDA grants Rare Pediatric Disease designations for serious or life-threatening diseases that primarily affect individuals aged from birth to 18 years. If approved, Prometic’s PBI-4050 could be eligible to receive a Rare Pediatric Disease Priority Review Voucher.

The vouchers can be used to reduce the time of an FDA new drug approval review to six months from ten months. The vouchers are potentially lucrative because they are transferable. Most recently, Ultragenyx and Kyowa Hakko Kirin sold their Priority Review Voucher for $80.6 million.

“We are pleased to have been granted this Pediatric Rare Disease designation by the FDA, which recognizes the rare and serious nature of EB and its impact on the lives of children suffering with this condition,” said Joe Wiley, CEO of Amryt. “With the interim efficacy readout for our phase 3 trial of AP101 in EB due later this year, we look forward to continuing to demonstrate progress with our lead development asset.”

August 7, 2018
Photo: Joe Wiley, CEO of Amryt