WHAT IS Systemic Sclerosis (SSC)?
Systemic sclerosis (SSc) is a rare auto-immune and severe disease which affects an estimated 170,000 patients in the US and the EU.
SSc is a progressive fibrotic disease which initially affects the skin and eventually spreads to other organs such as the blood vessels, heart, lungs, kidneys, gastrointestinal tract and the musculoskeletal system. Unfortunately, the fibrosis of inner organs leads to significant morbidity and mortality of patients with SSc: 50% of patients will die within 10 years after the first diagnosis*. Despite its severity, there is currently no cure or approved treatment for SSc although some symptoms are being treated with rheumatology or immunosuppressive drugs**.
The total market potential of SSc is estimated above $1.8bn.
* Eular SSc Trials and Research Group, EUSTAR, SSc Research Foundation, Canadian SSc research group.
LANIFIBRANOR – OUR SOLUTION
Lanifibranor is a small molecule activating all three subtypes of peroxisome proliferator-activated receptors (PPAR). PPARs are nuclear receptors involved in regulating fibrotic processes in the body. In several preclinical studies, lanifibranor has shown to reduce fibrosis in the organs affected in SSc: the skin, lung and kidney with positive effects on lung and heart function.
Lanifibranor is currently being studied in a Phase IIb clinical trial called FASST to treat diffuse cutaneous SSc (dcSSc). Enrolment has been completed for this trial and results are expected in early 2019.
HOW IS Systemic Sclerosis (SSc) DIAGNOSED?
Women are more commonly affected by SSc and are typically diagnosed in their 40s and 50s. Often, the first symptom is “Raynaud’s phenomenon”, a cold- and stress-induced swelling of fingers and hands along with discoloration of the skin, or digital ulcers, sores on fingers or toes caused by poor blood circulation.
Patients suffering from the limited form of SSc develop only hardened skin on hands, feet and the face whereas patients with the more severe diffuse form show skin thickening over a wider area of the body. The detection of auto-antibodies confirm SSc as an auto-immune disease.
To find a patient association, please visit the websites of the Federation of European Scleroderma Associations (FESCA) or The Scleroderma Foundation.