Amryt’s Epidermolysis Bullosa treatment granted Rare Pediatric Disease from US FDA

Amryt ensure US Food and Drug Administration’s grant a Rare Pediatric Disease designation for its lead development asset AP101, for the treatment of Epidermolysis Bullosa (“EB”).

Rare Pediatric Disease designations are granted for diseases that predominantly affect children aged 18 or younger and which affect a total of fewer than 200,000 people in the US. The designation means if a New Drug Application (“NDA”) for AP101 is approved, Amryt will be eligible to receive a priority review voucher that can be used, sold or transferred.


EB is a rare genetic skin disorder that 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 approximately 500,000 people living with EB worldwide and there are currently no approved treatments.2

AP101 is currently in a Phase III clinical trial (EASE), the largest ever global Phase III study for EB. An interim efficacy data readout is due later this year and the top-line data readout is expected in Q2 2019.


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

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