EUDRAC recently collaborated on a successful PRIME application with The EspeRare Foundation, a Swiss not-for profit Biotech Company which develops existing therapies to address severe unmet therapeutic needs in rare diseases. The application was for a protein replacement therapy (ER004) for X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED).
XLHED is an orphan disease which most significantly affects new-born males. Babies are born with absent or abnormal ectodermal structures including a lack of sweat glands. The condition is associated with significant morbidities and an increased risk of infant mortality.
Eligibility to PRIME was granted by the EMA on 13 October 2017 based on the seriousness of the disease and the recognition that it represents an unmet medical need for which there is a lack of available treatment.
EUDRAC is continuing to provide regulatory support for the development of ER004 and recently attended the PRIME kick-off meeting at the EMA together with representatives from EspeRare, the Coordinating Investigator and the President of the Ectodermal Dysplasias International Network. The meeting discussed the future development plan for ER004 and the support that will be provide by the EMA through the PRIME scheme.
ER004: Human immunoglobulin G1 constant region - human ectodysplasin-A1 receptor-binding domain fusion protein; ODD ; EMA/PRIME/17/053
The EspeRare Foundation: Founder – Dr Florence Porte; Address – Campus Biotech Innovation Park, Avenue Secheron 15, 1202 Genève, Switzerland