Maker of world's most expensive prescription drug flashes PhIII data for a potential fourth product
Three years before they became owners of the most expensive drug in the country, Amryt began its corporate life around a single therapy picked up from a small German drugmaker called Birken: Episalvan, an EMA-approved drug for partial thickness wounds. They hoped to develop it as an orphan drug for a condition called epidermolysis bullosa, where a patient’s skin becomes as fragile as butterfly wings. They put it almost immediately into a Phase III study.
Today, Amryt announced, the lead drug cleared the trial. Only a portion of topline results was released, but the Irish drugmaker said that across 223 patients, those who received the drug were more likely to see their wounds close within 45 days than those on placebo. The company didn’t say how much more likely, but noted that the P value was 0.013. Amryt said it was the first positive Phase III result for any drug in epidermolysis bullosa, and CEO Joe Wiley called it “another significant milestone” for the company.
The drug, though, missed on all key secondary endpoints — which most notably included how quickly the first wound healed after receiving treatment or placebo. The company added that “a number of favourable differences were observed” and more data would be coming out soon.
Amryt has already branded the epidermolysis bullosa version of the drug as Filsuvez. If approved, it would be the company’s fourth product, after Episalvan and two drugs they picked up last year from the bankrupt orphan drugmaker Aegerion. Those drugs, Myalept and Juxtapid, were noteworthy for consistently ranking among the most expensive drugs in the US, according to a list maintained by GoodRx. Amryt upped the price of both in January by 9.9% — keeping with an Aegerion practice — and Myalept now is ranked as the highest-priced drug.
The list does include those, like gene therapies, that patients receive in the clinic, although a separate report found Myalept was only behind Novartis’ $2 million gene therapy Zolgensma in total annual cost.
Companies do not traditionally release drug prices before approval and Amryt did not immediately respond to a request for comment. Orphan drugs, though, are traditionally among the highest-priced, fueled in part by limited competition. Around 1 in 20,000 people are estimated to have EB.
Although admittedly more a commercial company than a research one — “We’re not a research-based company, we don’t have labs anywhere,” Wiley told Endpoints News last year — the company does have one other treatment in the pipeline. In 2018, they in-licensed a gene therapy for epidermolysis bullosa.