The study’s independent data monitoring committee has concluded that children aged between 21 days and four years can take part in the programme.
Amryt is developing AP101 as a potential treatment for Epidermolysis Bullosa, where skin blisters to the touch.
The company’s chief executive, Joe Wiley, called the committee’s decision “a significant step forward”, adding it could help address an unmet need for a devastating condition, often present from birth.
“This development represents another milestone for Amryt as we build on our vision of becoming a global leader in rare and orphan diseases," he went on.
In a note to clients, analysts at ‘house’ broker Shore Capital said the update “demonstrates continued momentum at Amryt, following the significant positive interim efficacy update” at the start of January.
They added; “We continue to view Amryt as a compelling investment opportunity to gain exposure to a diversified rare disease platform, with a marketed drug for an ultra-rare disease, a further de-risked Phase III asset in an untapped $1bn market opportunity, and an attractive pre-clinical gene therapy platform.
“Our risk-adjusted sum-of-the-parts valuation suggests Lojuxta alone is worth c.20p/share, with the Phase III EB asset and early-stage pipeline not reflected.”
In early afternoon trading, Amryt Pharma shares were 0.3% higher at 15.55p.
-- Adds analyst comment, share price --