Amryt strikes gold with rare-disease breakthrough

It’s been a big month for Irish pharma. In December, Amgen swooped on Irish-based rare disease specialist Horizon Pharma in a €26.8 billion deal that was the largest in the pharma sector all year and one of the five biggest deals worldwide across all sectors in 2022. Now, just a week into January, another Irish rare disease drug developer has been acquired.

The money paid for Amryt may be a fraction of the Horizon price tag, but the premium it commanded in getting the deal over the line is a multiple of its more established peer.

Started less than eight years ago with a specific focus on rare diseases, Amryt’s big bet was that a drug it was developing, Filsuvez, could provide a first treatment for a genetic condition, Epidermolysis bullosa (EB), accelerating healing if not necessarily providing a cure.

Only 10 per cent of the 7,000 or so rare diseases afflicting humans have any treatment. EB was one of the forgotten 90 per cent.

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It means the skin of sufferers blisters at the slightest touch — even putting on a sock or sitting into a car, for instance. Known also as “butterfly skin” disease because of the fragility of the skin, Amryt founder Dr Joe Wiley notes that patients “need opiates to get through dressing changes because as the bandages are taken off the skin comes off with them”.

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Most patients are children. And another risk for those with EB is a particularly aggressive form of skin cancer.

Last summer, it delivered, securing European approval for the drug. Its efforts put it on the radar of Chiesi Farmaceuti, a long-standing family-owned specialist in rare diseases, over the weekend which culminated in a €1.18 billion cash buyout of the Irish business.

A 107 per cent premium to the last price at which the company’s shares traded on the US Nasdaq index means it makes wealthy men of the firm’s founders and early investors. Moreover, this gives Amryt greater heft to market its EB and other therapies.

And there may be more to come. Chiesi has held out the prospect of another $225 million in milestone payments for Amryt’s investors. That will likely depend largely on persuading the US authorities to sanction the drug for that market. Amryt has triggered a formal dispute resolution request with the Food and Drug Administration to address scientific differences over the drug’s potential. A lot rests on the outcome.