The first medication specifically developed to treat a rare and painful skin condition ushers in an era of hope, according to charity Debra Ireland.
Epidermolysis bullosa (EB) causes the skin, both inside and out, to blister and wound at the slightest touch and the only treatment for the distressing condition is painful bandaging to prevent infection.
Filsuvez gel, developed by Dublin-based Amryt Pharma, has completed phase three clinical trials and shows that it can speed up wound closure and reduce the pain of bandaging.
Debra Ireland, which supports Irish EB patients and their families, said the new treatment could pave the way for game-changing therapies in the coming years.
Jimmy Fearon, Debra's CEO, said: "With a slew of exciting medical breakthroughs in the pipeline, for the first time ever the future is looking much brighter for people with EB.
"We believe that Filsuvez, the first of its kind to specifically treat EB, will open the door to a series of exciting and game-changing therapies that we expect to see in the next decade, with each one improving wound closure.
"That improvement will reduce the pain and trauma associated with bandaging, as well as the constant threat of infection that is always present with open wounds.
"Phase three trials are required for medicine approval from regulatory bodies around the world like the FDA (Food and Drug Administration) in the US or the EMA (European Medicines Agency) in Europe."
Amryt has reported that Filsuvez showed a 72% increase in the probability of wound closure compared with the control gel in patients who have recessive dystrophic EB, one of the most severe EB sub-types - for which no treatment currently exists.
Emma Fogarty, who lives with the disease, said an effective treatment for EB would be "the most amazing thing".
The 36-year-old from Co Laois said: "I could live, I could gain back some independence. I could get out and meet friends and go for a drink without worrying about being in pain.
"Something as small as finding a new bandage or a new painkiller that will work would make a difference. It doesn't have to be the cure. It just needs to help us in some way, and that would be life-changing."
Amryt also reported a greater reduction in pain associated with dressing changes.
Amryt Pharma CEO Dr Joe Wiley said: "These results represent a potentially important advancement for patients and families living with this rare and distressing disorder."
