LONDON (Alliance News) - Amryt Pharma PLC said Thursday that the US Food & Drug Administration has granted a rare pediatric disease designation for AP101, used for the treatment of Epidermolysis Bullosa
Epidermolysis Bullosa 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.
Amryt Pharma's AP101 is currently in a Phase III clinical trial and an interim efficacy data readout is due later this year and the top-line data readout is expected in second quarter of 2019.
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.1 The 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.
Shares in the company were trading 4.8% higher at 19.13 pence each on Thursday afternoon.
