EspeRare Receives FDA Breakthrough Therapy Designation for Investigational Prenatal Treatment ER-004 in X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED)

GENEVA, Switzerland I July 6, 2020 IEspeRare, a not-for-profit organization dedicated to accelerating the development of treatments for rare diseases, 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 Medicine1.

The FDA Breakthrough Therapy Designation intends to expedite the development and review of drugs for serious or life-threatening conditions. To be granted, it requires preliminary clinical evidence demonstrating that the drug may result in substantial improvement on at least one clinically-significant endpoint compared to available therapy. This Designation allows a program to benefit from all of the FDA's fast-track program features, including proactive FDA guidance on an optimal drug development plan, organizational commitment involving FDA senior managers, and eligibility for rolling and priority reviews2.

"Rare diseases such as XLHED often don't receive the attention and research investments that patients and their families deserve. The FDA's decision to grant Breakthrough Therapy Designation to ER-004 for the prenatal treatment of XLHED represents a significant milestone for patients and EspeRare alike in highlighting the severity of this disease and the importance of offering a potential first therapeutic option to this underserved patient population," said Caroline Kant, EspeRare's Executive Director, and added, "This innovative treatment approach has the potential to fundamentally change the lives of these patients and may also pave the way for other prenatal treatments to correct genetic diseases before birth. We look forwards continuing to working closely with the FDA and the XLHED patient community towards these goals."

Mary Fete, Executive Director of NFED (National Foundation for Ectodermal Dysplasias), commented: "This is extraordinary news for our Ectodermal Dysplasias community in the United States and around the world. The NFED led the effort for three decades to develop a treatment for XLHED. Having the FDA Breakthrough Therapy Designation means scientists can aspire starting a clinical trial to confirm the findings for ER-004 and ultimately, if successful, make this treatment an option for families. It brings us closer to an amazing time when children born with XLHED could have working sweat glands! We are honored to work with EspeRare to make this happen."

In the second half of 2021, EspeRare anticipates to start enrolling patients into a pivotal study. In the United States this program also benefits from Fast Track and Orphan Drug Designation. In Europe, the program receives support from the EMA's PRIME (Priority Medicines) and also the Orphan Drug Designation. With these multiple regulatory incentives, the program can aim at a streamlined worldwide development.

SOURCE: EspeRare Foundation

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