Amryt’s AP101 EASE Phase III trial has been extended to include infants aged 21 days to four years, marking a huge step forward in the search for an Epidermolysis Bullosa (EB) treatment in children.
An Independent Data Monitoring Committee (IDMC) allowed the extension after analysis was conducted using pharmacokinetic (“PK”) data received from patients already enrolled in the trial.
Amryt will begin the recruitment process for infants and children into EASE immediately. The EASE trial is the largest ever global Phase III study conducted in patients with EB and Amryt expects the top-line data read out in the second half of 2019.
Joe Wiley, chief executive of Amryt Pharma, commented: “Extending the age eligibility criteria for this trial is positive news and a significant step forward in addressing the unmet need in this devastating condition, which is present from birth in most cases. This development represents another milestone for Amryt as we build on our vision of becoming a global leader in rare and orphan diseases.”
Back in January the IDMC recommended that the trial should continue with an increase of 48 patients in the study, creating a total of 230 evaluable patients, in order to achieve 80% statistical power.
EB is a devastating, rare genetic skin disorder that can cause skin to blister and tear from the slightest friction or trauma, and there are approximately 500,000 people living with EB worldwide with no approved treatments.
The EASE trial is the largest ever global study to investigate a treatment for EB, and the continuation and extension means there fortunately there is real hope that the results show that the treatment is efficacious and ultimately works.