Amryt Pharma wins key US drug designation

FDA move means Irish company could receive voucher ultimately worth up to €300m

Irish pharmaceutical company Amryt Pharma has won a designation for one of its key drugs which could make it eligible for a voucher ultimately worth up to $350 million (€300 million).

The Dublin-based company has been granted a “rare paediatric disease designation” from the US Food and Drug Administration (FDA) for its lead development asset – a drug for the treatment of Epidemolysis Bullosa (EB).

EB is a rare genetic skin disorder that can cause skin to blister and tear from the slightest friction or trauma and can, in some cases, cause blistering and erosion of the lining of internal organs. Some 500,000 people live with the disease which currently has no approved treatments.

If Amryt’s new drug application is approved, it will be eligible to receive a priority review voucher that can be used, sold or transferred.


Earlier this year Jazz Pharmaceuticals paid Spark Therapeutics $110 million for such a voucher. The highest price paid for a priority review voucher is $350 million in August 2015 when United Therapeutics sold its voucher to AbbVie.

The vouchers are, however, usually reserved for badly needed drugs for serious diseases and shave off around four months of the FDA review period for any drug.

Trial results

Amryt’s drug, AP101, is currently in a phase three clinical trial with important results from the trial expected in the second quarter of 2019.

"We are pleased to have been granted this Paediatric Rare Disease designation by the FDA, which recognises the rare and serious nature of EB and its impact on the lives of children suffering with this condition," Amryt chief executive Joe Wiley said.

Aside from pushing to develop a drug for EB, Amryt works to deliver new treatments primarily for rare or orphan diseases. Its Lojuxta treatment for adult patients with a rare cholesterol disorder is already on the market.

The Lojuxta treatment was last month approved for funding as an NHS England treatment for adults. The decision means that, from this year, patients with the life-threatening genetic condition being treated on the NHS in England can be prescribed the medicine, which can reduce the production and uptake of bad cholesterol.

Peter Hamilton

Peter Hamilton

Peter Hamilton is a contributor to The Irish Times specialising in business