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.
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!
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