Pierre Fabre pursues its commitment against rare diseases in the pediatric, oncology and dermatology fields.

RARE DISEASE DAY – February 28th,2023

The diseases are rare,but they impact a lot of people : nearly 300 million people are affected worldwide1.

CASTRES,France,Feb. 6,2023 --International Rare Disease Day® will be held on February 28,an opportunity for the Pierre Fabre Group to reiterate its commitment in this area,in which it is essential to put concrete action plans in place covering the entire spectrum of care from diagnosis to treatment and access to quality health care.There are around 5,000 to 8,000 known rare diseases in the world today2,95% of which do not yet have a treatment3.

In addition to oncology and dermatology,Pierre Fabre has been involved in pediatric diseases for several years now,developing specific therapies for that patient population,which requires adapted treatments. Examples include infantile hemangioma where a therapy has been approved since 2014 and,more recently,X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED) with a therapy under development.

Finding a treatment for children with XLHED,with the EspeRare Foundation

The Pierre Fabre Group has recently committed,with the EspeRare Foundation,to the development of an innovative therapy for the most common type of Ectodermal Dysplasia,XLHED,a rare genetic disease that affects the sweat and respiratory glands,skin,hair and teeth. EDELIFE,a large-scale clinical study,involving women who are pregnant with a boy carrier of the disease gene,was launched in July 2022.

For more information:

https://edelifeclinicaltrial.com/

Article from the New England Journal of Medicine

"I hope he gets to have a more normal life than my dad. That he's not restricted,that he can get to play outside with his friends. I want him to be able to accomplish what he wants and not have that hurdle (of XLHED)."

"I feel so much gratitude for the people doing the research and putting things in place."

Emily,mother of a boy affected by XLHED and treated in utero.

For more information on the Pierre Fabre Group's other commitments in the fight against rare diseases,please consult the complete press release in the media area of the company's website : https://www.pierre-fabre.com/en/mediaroom

1https://www.rarediseaseday.org/

2 https://www.ema.europa.eu/en/human-regulatory/overview/orphan-designation-overview

3https://www.thelancet.com/journals/landia/article/PIIS2213-8587(19)30006-3/fulltext

Press contact:

Anne Kerveillant

anne.kerveillant@pierre-fabre.com