[{"orgOrder":0,"company":"EspeRare","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"EspeRare Gets FDA Breakthrough Therapy Designation for ER-004 in X-Linked Hypohidrotic Ectodermal Dysplasia","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"SWITZERLAND","productType":"Large molecule","productStatus":"New Molecular Entity","date":"July 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II"},{"orgOrder":0,"company":"EspeRare","sponsor":"Pierre Fabre","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"The EspeRare Foundation and Pierre Fabre Join Forces to Develop and Market a Pioneering Treatment for XLHED","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"SWITZERLAND","productType":"Large molecule","productStatus":"New Molecular Entity","date":"December 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I"},{"orgOrder":0,"company":"EspeRare","sponsor":"Pierre Fabre","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Pierre Fabre and The Espe Rare Foundation Start The EDELIFE Clinical Trial of A Prenatal Treatment for A Rare Genetic Disease, XLHED","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"SWITZERLAND","productType":"Large molecule","productStatus":"New Molecular Entity","date":"November 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II"},{"orgOrder":0,"company":"EspeRare","sponsor":"Pierre Fabre","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Pierre Fabre and the EspeRare Foundation Administer Investigational Treatment to First Patient in EDELIFE Clinical Trial for Rare Genetic Disease, XLHED","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"SWITZERLAND","productType":"Large molecule","productStatus":"New Molecular Entity","date":"July 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II"}]
Find Clinical Drug Pipeline Developments & Deals by EspeRare
ER004 is a pioneering 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 foetus.
The EDELIFE clinical trial will investigate the efficacy and safety of intra-amniotic ER004 as a prenatal treatment for male foetuses who have been confirmed to have XLHED.
The Pierre Fabre group will be granted exclusive worldwide rights for the development, manufacturing and commercialization of ER-004. EspeRare and the Pierre Fabre Group will pool their respective expertise together in order to co-develop ER-004.
The Breakthrough Therapy Designation was granted following the promising results from three XLHED subjects who were treated by Professor Holm Schneider with a course of ER-004 intra-amniotic injections during the third trimester of pregnancy.