BRIEF — Rare Pediatric Disease designation for Amryt's AP101

The US Food and Drug Administration (FDA) has granted a Rare Pediatric Disease designation for AP101, Amryt Pharma’s lead development asset, for the treatment of epidermolysis bullosa (EB.)

This designation means if a New Drug Application for AP101 is approved, Amryt will be eligible to receive a priority review voucher that can be used, sold or transferred.

AP101, which has already been approved in Europe for use in the treatment of partial thickness wounds in adults, is currently in a Phase III clinical trial, 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.