Amryt on track with skin treatment for butterfly children

Amryt is on track to begin the phase 3 trial at the end of March for its new skin healing treatment in an orphan disease, with regulatory authority discussions now completed with Food and Drug Administration (FDA) and European Medicines Agency (EMA) and the design of the clinical trial established.

Amryt, the pharmaceutical company focused on treatments for rare and orphan diseases, has been in talks with the FDA and EMA about the design of its pivotal phase 3 clinical trial for AP101 as a potential treatment for Epidermolysis Bullosa (EB).

EB is a rare genetic skin disorder that leads to exceptionally fragile skin and children with the disorder are often referred to as “Butterfly Children”. The global market for a treatment in EB is estimated to be in excess of €1.3 billion, Amryt noted.

Amryt has also agreed to conduct some further non-clinical studies in parallel with this phase 3 study. INC Research has been appointed as the contract research organisation for the phase 3 study, and approximately 30 clinical trial sites in 15 countries have already been pre-qualified.

Joe Wiley, Chief Executive Officer of Amryt, said that reaching agreement with the regulatory authorities for the phase 3 study in EB was a precursor to start the pivotal study.

“Significantly, we now have agreement from the regulatory agencies in both the US and in Europe for a single phase 3 study, which we hope in due course will lead to approval in EB. There remains substantial unmet need for drugs which can address the symptoms and we are delighted to be progressing our potential treatment to this pivotal phase,” said Wiley.