Amryt gets American regulatory boost

Shares in Irish-listed speciality drug firm Amryt Pharma rose by 4% yesterday on the back of the company gaining orphan drug designation in the US for one of its compounds.

Amryt, which effectively relaunched itself earlier this year via the reverse takeover of Dublin investment firm Fastnet Equity, yesterday announced the Food and Drug Administration (FDA), in the US, has granted orphan drug designation to its AP102 compound, which has the potential to treat patients suffering from acromegaly — a rare disorder resulting in abnormal body growth due to the body producing excessive growth hormone.

Acromegaly affects up to 13 in every 100,000 people in the US.

Orphan diseases are traditionally described as rare conditions that affect fewer than 200,000 people in the US and orphan drug designation (via the US Orphan Drug Act) aims to bring such drugs to market in greater numbers by offering certain financial benefits to manufacturers.

Via this designation, Amryt will qualify for tax credits and market exclusivity upon regulatory approval.

Amryt’s chief executive Joe Wiley yesterday described the development as “an important regulatory milestone” for the company ahead of it starting clinical trials of AP102 next year.

Two months ago Amryt was granted a US patent for its lead drug, Episalvan, to be used as a treatment for the rare skin disorder epidermolysis bullosa (EB).

Amryt is aiming to be the world leader in EB treatment, a sub-market of the wider orphan care sector estimated to be worth around €1.3bn annually.