Scleroderma, also known as systemic sclerosis (SSc), is a rare, progressive, debilitating autoimmune disorder characterized by skin fibrosis, systemic inflammation, and vasculopathy that manifests as Raynaud’s disease, skin thickening, and, often, painful digital ulcers. Mortality risks of pulmonary arterial hypertension (PAH), interstitial lung disease (ILD), and renal crisis are high in SSc patients. The only disease-modifying therapies (DMTs) approved for SSc are Boehringer Ingelheim’s Ofev (nintedanib) and Roche / Genentech’s Actemra / RoActemra (tocilizumab), both of which target the SSc-ILD subpopulation and have demonstrated the ability to slow the progressive loss of lung function. Other therapies, such as bosentan (Actelion’s Tracleer, generics) and Bayer’s Adempas (riociguat), are commonly prescribed for overlapping symptoms of PAH. The SSc pipeline is large and diverse, but most agents are in earlier phases of development. Although tremendous need exists for additional effective DMTs, drug development in SSc has been historically challenging owing to the heterogeneity of SSc patients and the commensurate challenges with trial design.
Niche & Rare Disease Landscape & Forecast provides comprehensive market intelligence with world-class epidemiology, keen insight into current treatment paradigms, in-depth pipeline assessments, and drug forecasts supported by detailed primary and secondary research.
Niche & Rare Disease Landscape & Forecast introduces a new Drug Pipeline chapter with real-time, global pipeline intelligence content directly from Cortellis. This chapter is updated daily and features interactive figures that can be easily downloaded for detailed analysis or presentations.