Amryt Pharma said Filsuvez, its topical therapeutic gel, had demonstrated a significant increase in speed of wound healing in the rare disease Epidermolysis Bullosa (EB) in a late-stage trial.
This would be the first treatment to do so.
The company said today that US regulatory submissions were already underway with the Food and Drug Administration (FDA), with a request planned for priority review.
EU regulatory submission was also planned with a request for accelerated assessment, it added.
EB is a rare, chronic and distressing genetic skin disorder that causes the skin layers and internal body linings to separate and affects infants, children and adults.
The global incidence of EB is estimated to be approximately one in 20,000, which implies that there are as many as 30,000 affected individuals in the US and over 500,000 worldwide.
There are currently no approved treatments.
The global market opportunity for EB is estimated by the company to be in excess of $1 billion.
Dr Joe Wiley, CEO of Amryt Pharma, said the positive outcome of the Phase 3 EASE trial marks another significant milestone for Amryt as it seeks approval for Filsuvez.
He said it represents a potentially important advancement for patients and families living with this rare and distressing disorder.
"If approved, we intend to leverage our existing global infrastructure to commercialise Filsuvez," he added.
