After a challenging year, we are excited to share some good news – just in time for the holidays!
The EspeRare Foundation announced a new partnership today that brings us closer to developing a potential treatment for x-linked hypohidrotic ectodermal dysplasia (XLHED). EspeRare and Pierre Fabre group have entered into an agreement to develop and commercialize ER-004, a pre-natal treatment for XLHED.
Next Step: The Clinical Study
They plan to launch the next clinical study in 2021. The purpose is to confirm findings from Prof. Holm Schneider’s study at the University of Hospital Erlangen in which he treated three babies in-utero with ER-004.
Those babies developed normal working sweat glands and had other improved symptoms, which you can learn more about. His findings were published in the New England Journal of Medicine, the British Journal of Clinical Pharmacology and Nature Medicine’s Research Highlights. The upcoming study would treat a larger number of babies using the in-utero therapy in late pregnancy.
If successful, EspeRare and Pierre Fabre aim to have ER-004 as the first approved treatment for XLHED on the market by 2026. It’s extraordinary to think about!
Grateful for These Collaborators
The National Foundation for Ectodermal Dysplasias (NFED) has been proud to collaborate with the executive director of the Swiss nonprofit EspeRare, Caroline Kant, and her team since they acquired ER-004 in 2017. Their new partner, Pierre Fabre, is the second largest private pharmaceutical group in France and the second largest dermo-cosmetics laboratory in the world.
We are thrilled to learn that Pierre Fabre has come on board to develop this potential treatment. With this long established pharmaceutical company in place, the clinical study can begin sooner rather than later.
All of us at the NFED are grateful to both companies for their dedication to helping our underserved population of individuals affected by this rare disorder. It brings incredible hope for our NFED community, and especially our XLHED families, to know that a therapy could be on the market in the next five years!
Decades in the Making
The NFED started this XLHED research in the late 1980s and has continued to be its driving force through its ups and downs over the years. See XLHED research timeline.
Developing a treatment has been our goal for decades. The NFED is committed to supporting EspeRare and Pierre Fabre with the clinical study in any way that we can.
Mary Fete
Together, we can work toward a future where XLHED families have a treatment that gives them normal sweat glands! With the FDA breakthrough therapy designation in the United States and this partnership, it’s exciting to be closer to this reality.
Co-Developing ER-004
According to the agreement, the two groups will co-develop ER-004. The Pierre Fabre group will have the exclusive worldwide rights for the development, manufacturing and commercialization of ER-004.
Kant said, “At EspeRare, we are thrilled to collaborate with the Pierre Fabre Group to co-develop this very inspiring program. We are confident that joining forces with Pierre Fabre will allow to bring this ground-breaking therapy to XLHED patients in alignment with our patient-centered values.”
We are proud to work hand in hand with the NFED and the XLHED community at large, thriving to improve the lives of patients and their families.
– Caroline Kant
According to Eric Ducournau, CEO of the Pierre Fabre Group, “We are proud to ally with the EspeRare foundation to find a therapeutic solution to a debilitating disease affecting 500 babies worldwide every year. We cannot think of a better way to epitomize our corporate purpose: ‘Every time we take care of one single person, we make the world better.’”
The world of research and development always takes time – and patience. That’s why the NFED has persisted through the ups and downs. News like today’s gives us incredible hope for the future of XLHED!
Read EspeRare’s Press Release
Ectodermal dysplasia can cause a lifetime of challenges. By supporting research, you expand early diagnostics, treatments, pathways toward cures… and hope!Shape Our Futures With Research
Is this only for babies and babies in-utero? or will it be tested on other children for example my daughter 7 year old who was diagnosed with HED?
This treatment is administered to babies in-utero who are affected by the x-linked form of HED. Unfortunately, we know from a past clinical study that the treatment does not work if given after birth. The protein must be given in-utero to affect the development of the various body parts to work normally. We are so sorry that this will not help your daughter. ~ Jodi, NFED, Director, Marketing and Communications
Wonderful NEWS for those considering having children and for the future generations with an XLHED parent or grandparent. I praise our foundation for the foresight research in the early years of development and for the continued support for research. Mother of a 37 yr old male with XLHED.
Hi, Pam! We are glad to hear you are celebrating this wonderful news with us. Your family was one of those first families who believed in the NFED’s research efforts and volunteered to move it forward. Those efforts have led us to the progress today. And you are so right when you say..”our foundation” because that’s what is, the families’ foundation. Thank you for your role in this success! Sending you and those boys of yours our best. ~ Jodi, Director, Marketing and Communications
I am so excited to read about ER-004. Even though our Son will not benefit directly from this, it may help his Grandchildren. What a blessing to people having families now. Our son who is 44 now, was born with XLHED, but was not diagnosed until he was about 9 months. We were out of town and he began to run a fever again. We went to the ER and the pediatrician there had see it one other time when he was in the service. We were thankful that he ran a fever that day, or it would have been much longer before we knew why he was running fevers, and had skin issues most of the time. We even went to Mayo Clinic in Rochester after Dr. Fane told us about E.D, and Mayo had not heard of it or seen it. Once diagnosed, we could make adjustments at home to make life much better for all of us. He has lived life with no limitations placed on him other than what he set for himself. He figured out ways to keep cooler and still be outside with friends in the summer growing up, and still spends a lot of time out baling hay in the summer. We found out about NFED when he was 2, and the foundation was just starting. Thank You NFED, and all who partnered with you. for the brighter future for families that would be affected by this.
Peggy, thank you for sharing with us. We’re happy to know that you have been on this journey with us!
I was of the view that the treatment of in utero baby had already been introduced by your reports earlier so what’s the new research.
Good day and thank you for reaching out. Dr. Schneider continues his work but no official clinical trials have not yet started. EspeRare and Pierre Fabre group have entered into an agreement to develop and commercialize ER-004, a pre-natal treatment for XLHED. This is a new partnership. Stay tuned for updates!