Amryt Pharmaceuticals DAC: Filsuvez

We acknowledge receipt of your major amendment dated September 27, 2021, which extended the goal date by three months.

(1) The submitted data do not provide substantial evidence of effectiveness for the use of Filsuvez (birch triterpenes) gel for the proposed indication of treatment of wounds associated with inherited epidermolysis bullosa (EB).

Although the primary endpoint for Study BEB-13 was met (Filsuvez treatment resulted in an increased proportion of subjects with first complete closure of the EB target wound within 45 days relative to control gel), the pre-specified and exploratory secondary endpoints provide limited support for a substantial benefit with active treatment.

Due to their design and significant amounts of data unavailable for blinded assessment, these studies are not suitable for providing confirmatory evidence.

The short-term in vitro studies referenced in this application used cells cultured from healthy volunteers; there are no data on the drug's effect on the basement membrane defects in EB.

Thus, we have concluded that there are insufficient data in your application to support a determination that Filsuvez treatment reliably results in accelerated wound healing or reduced total wound burden.

To address this concern, submit additional confirmatory evidence of effectiveness of your product for EB or specific subsets of the EB population.