LONDON (Alliance News) - Amryt Pharma PLC on Thursday said it now has permission to enrol infants and children under four years old in its ongoing phase three Ease trial of AP101 in the US.
The Ease trial will assess AP101 as a treatment for epidermolysis bullosa, a rare genetic skin disorder that can cause the skin to tear and blister easily and can also damage the lining of the body's internal organs.
Amryt is an orphan drug company, meaning it develops drugs for rare diseases such as epidermolysis bullosa, and will now be able to enrol patients as young as 21 days old onto the trial.
Permission to enrol young children and infants in the trial was granted after an assessment by an independent data monitoring committee, which looked at data from patients over four years old who are already involved in the trial.
Amryt will commence recruitment for infants and children to join its trial immediately. To-line data from Ease is expected in the second half of 2019.
Ease is the largest ever global phase three study of epidermolysis bullosa patients. Amryt estimates that the market potential for AP101 surpasses EUR1 billion.
"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," said Amryt Chief Executive Joe Wiley.
Shares in Amryt were trading flat at 15.50 pence on Thursday.
