Pharmaceutical giant Amryt has been given the green light by European and US regulators to carry out trials of a new drug to treat a rare skin disease.
The genetic condition known as Epidermolysis Bullosa (EB) makes the skin of those afflicted extremely delicate.
Children suffering from the disease are known as "Butterfly Children".
The global market for treatment of EB is thought to be around €1.3bn annually.
After completing discussions with the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA), Amryt is on track to commence a phase 3 trial at the end of March. The company has agreed to conduct some further non-clinical studies in parallel with the phase three study.
"We are delighted to have reached this critical milestone in the development of Amryt," said Amryt ceo Joe Wiley.
"Significantly, we now have agreement from the regulatory agencies in both the US and in Europe for a single phase three study, which we hope in due course will lead to approval in EB," Mr Wiley added.
INC Research has been appointed as the contract research organisation for the phase three study. Approximately 30 clinical trial sites in 15 countries have already been selected.
Adult and paediatric patients with EB will be enrolled into a random placebo controlled trial. A total of 164 patients will be treated for a 90-day period.
"We believe that the adaptive approach to study sample size will help to ensure that we achieve a reliable assessment of the potential benefit of our new skin healing treatment in this orphan disease," said Mark Sumeray, chief medical officer at Amryt
Last month, Amryt announced it had received a patent for its lead drug Episalven in Japan. The company received substantial funding last year from Dublin-based investment group Fastnet.
The deal saw Harry Stratford, founder of pharmaceutical giant Shire, join the Amryt board as non-executive chairman.
Amryt also appointed former Sudocrem founder Ray Stafford to the board.
Mr Stafford said on his appointment that he expected Amryt's new drugs to yield around $1.5bn over the course of their lifetime.
The diversity of the Amryt board is testament to the possibilities that may lie ahead in the treatment of EB and other rare diseases.
The Amryt/Fastnet deal is believed to have been the brainchild of Cathal Friel, a founder of Raglan Road Capital, who serves as a non-executive director.