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EspeRare & Pierre Fabre | A pioneering treatment for XLHED

14.12.2020
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Co-develop the ER-004 treatment

 

The EspeRare Foundation and Pierre Fabre join forces to develop and market a pioneering treatment for XLHED, a dermatologic-related rare genetic disease that requires prenatal therapeutic intervention.  They announced today that they have entered into a license and development collaboration agreement for the development and commercialisation of ER-004, a prenatal treatment for XLHED (X-linked Hypohidrotic Ectodermal Dysplasia), a rare, debilitating congenital disease. The next clinical study is expected to start in 2021 and will aim at qualifying and registering what may become the first approved treatment for XLHED by 2026.

According to the terms of the agreement, EspeRare and the Pierre Fabre Group will pool their respective expertise together in order to co-develop ER-004. The Pierre Fabre group will be granted exclusive worldwide rights for the development, manufacturing and commercialisation of ER-004.

 

THE ESPERARE FOUNDATION

EspeRare is a Swiss non-profit organisation founded in 2013 that is committed to improve the lives of children with life-threatening rare diseases. EspeRare addresses the unmet medical needs of these children by uncovering the potential of existing treatments. EspeRare’s innovative model combines pharmaceutical know-how with philanthropic, public and private investments to develop and bring to life these discontinued therapies. With its unique patient-centered approach to drug development, EspeRare engages the patient community at each step of the process, with the intent of giving children and their families fair access to these therapies and a new hope for the future

 

XLHED

XLHED is a genetic disorder that affects the structure of the ectoderm, the most exterior part of the three primary germ layers formed during early embryonic life, from which the skin and its appendages are derived. ER-004 is pioneering an in-utero therapy designed to replace the function of endogenous Ectodysplasin A1 (EDA1), a protein key to the normal development of ectodermal structures in the fetus. ER-004 is a recombinant, soluble, and humanised form of EDA1 that is given as a single course treatment and delivered through intra-amniotic injections during the late stage of pregnancy. This approach has already demonstrated a significant potential in humans where it normalised sweat gland function in three patients treated in this fashion by Prof. Holm Schneider at the University Hospital Erlangen in Germany. First results were published in the New England Journal of Medicine1 and in the British Journal of Clinical Pharmacology2 as well as featured in Nature Medicine’s Research Highlights3.

 

Full Press Release.