Repurposing of losartan for treatment of epidermolysis bullosa

Abstract

Introduction & Objective

Losartan, a well-established medication to treat hypertension, has shown promise for the treatment of dystrophic epidermolysis bullosa (DEB), a hereditary rare skin blistering disease with secondary scarring and fibrosis. In a mouse disease model of dystrophic EB, losartan reduced fibrotic scarring and prevented fusion of digits.

Our objective is to generate additional required data on the benefit of losartan in the treatment of DEB. We aim to obtain regulatory approval for losartan for the treatment of DEB.

Materials & Methods

A multidisciplinary team of physicians, pharmacists and entrepreneurs has come together and bundled efforts in the start-up company Crowd Pharma. The new ‘virtual’ company is dedicated to repurpose losartan for the treatment of DEB.

Results

A ‘second-medical-use’ patent of losartan for the treatment of EB has been granted in Europe (pending in the US).

A new pediatric, easy-to-swallow formulation of losartan as minitablet has been developed that is suited for treatment of DEB (patent pending)..

A first clinical trial of losartan in EB, funded by patient organisation DEBRA, was successfully completed at the EB center in Freiburg in 2021.

Orphan designation has been granted by regulatory agencies EMA and FDA, and protocol assistance / pre-IND advice procedures have been completed.

Conclusions

Currently available evidence of efficacy and safety of losartan for the treatment of DEB is not considered sufficient by regulatory agencies. A placebo-controlled randomized clinical trial is required, for which fundraising is ongoing.