Amryt data strengthen the case for approval

The unveiling of more pivotal data for Amryt Pharma's Filsuvez has only deepened the mystery as to why this epidermolysis bullosa project managed to hit its complete wound closure primary endpoint but missed on a 90-day target wound closure secondary endpoint. However, progression to approval is unlikely to be halted, given what looks to be compelling efficacy on the important primary measure at the 45-day point. And apparent efficacy, albeit post hoc, hit in a subset of patients with recessive dystrophic forms of the disorder could strengthen the case for a regulatory green light. For 175 patients with recessive dystrophic EB the difference in complete closure of the target wound within 45 days was 44.0% for Filsuvez against 26.2% for placebo (nominal p=0.008). Add to this that an apparently narrow miss in the pain endpoint and the lack of treatments for this distressing skin disorder, and the approval case becomes even more compelling. Amryt itself is wasting no time and is moving full steam ahead with filings in both the US and Europe, and expects to complete these by the end of the first quarter.

Ease trial 
Primary endpoint, measured at day 45 Oleogel-S10  Control  P value
    % of patients with complete closure of EB target wounds 41.3% n=104 28.9% n=114 0.013
Secondary endpoints, measured at day 90 Oleogel-S10  Control  P value
    % of completely closed target wounds  55.5% n=104 43.9% n=114 0.296
    Reduction in procedural pain from baseline  1.32 n=76 0.18 n=78 0.051
    Reduction in total body wound burden 3.4 n=84 2.8 n=85 NS
    Reduction in total body surface area of EB partial thickness wounds 4.32 n=86 2.53 n=85 NS
NS=not significant. Source: company data. 

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