Rare diseases
Millions of patients worldwide suffer from rare diseases, which have become a leading health issue. In keeping with our company purpose, we are actively involved in this field to address unmet medical needs.
Our new field of excellence
In Europe, a disease is considered to be rare if it affects fewer than one out of every 2,000 people. While rare disorders affect a small number of individuals, they affect a significant number of patients overall on a global scale, across all regions of the world. To drive forward the discovery and development of new treatments, building strong partnerships is essential.
- 200 million children worldwide suffer from a rare disease
- 8,000 rare diseases identified worldwide
Infantile hemangioma
Infantile hemangioma - benign tumors of the blood vessels - affect 4.5% of infants. In partnership with Bordeaux University Hospital, we developed the first scientifically approved pediatric drug to treat this disorder.
XLHED
With the EspeRare Foundation, since 2021, we have been engaged in a rare form of ectodermal dysplasia known as XLHED, which affects a number of infant boys in Europe and the USA every year. We believe that every life matters, regardless of the number of patients concerned.
EBV+ PTLD
EBV PTLD (Epstein-Barr-positive post-transplant lymphoproliferative disease) is a rare type of lymphoma that can affect immunosuppressed patients after a solid organ transplant or hematopoietic stem cell transplantation. There is a significant unmet need for patients who are not receptive to first-line treatments, because they only have a survival rate of a few weeks or months after those treatments fail, and other treatment options are limited.
A therapeutic breakthrough honored by the Prix Galien International Award
Laboratoires Pierre Fabre received the ‘Prix Galien International’ Award in 2024 in the ‘Best product for a rare/orphan disease’ category. By awarding this prestigious distinction to Laboratoires Pierre Fabre, the jury endorsed an innovative manufacturing process and mechanism of action. The award represents powerful global recognition of Laboratoires Pierre Fabre commitment to patients who suffer from or have suffered from cancer.
Ichthyosis
Ichthyosis is a rare genetic skin disorder that manifests in a variety of forms, with symptoms that range from mild to severe, and can have a big impact on quality of life, because it significantly affects the skin barrier.
We led a global study called ALL, assisted by four European ichthyosis patient associations and three world-renowned specialists, to better understand their needs and experience. For the first time, nearly 400 patients got a chance to share their experience: 44% reported that the disorder affected their face, 51% their hands, making it highly visible and aggravating the impact on their social life. 85% confirmed that the disorder affected their personal life. Those findings were published in 2024 in the British Journal of Dermatology.
May El Hachem, Rita De Marco, José María Soria de Francisco, Anne Audouze, Mandy Aldwin-Easton, Charbel Skayem, Charles Taieb, Markéta Saint Aroman, Hélène Ghienne, Catherine Baissac, Juliette Mazereeuw, Christine Bodemer, Ichthyosis: multinational European study on patient characteristics, involved body sites and impact on quality of life, British Journal of Dermatology, Volume 190, Issue 5, May 2024, Pages 773–775, https://doi.org/10.1093/bjd/ljae082
