Posted by Michael Wonder on 06 Jul 2020
EspeRare receives FDA breakthrough therapy designation for investigational prenatal treatment ER-004 in X-linked hypohidrotic ectodermal dysplasia
6 July 2020 - EspeRare today announced that the U.S. FDA has granted breakthrough therapy designation to investigational ER-004 protein replacement therapy for the treatment for the prenatal treatment of XLHED, a life-threatening rare genetic condition.
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.
These results, demonstrating that this prenatal treatment has a profound and life-changing effect on these infants, were published in the New England Journal of Medicine.
