Scleroderma (systemic sclerosis [SSc]) is a rare progressive autoimmune disorder characterized by skin fibrosis, systemic inflammation, and varying degrees of vasculopathy that manifest as Raynaud’s disease and, often, painful digital ulcers. Risks of pulmonary arterial hypertension (PAH), interstitial lung disease (ILD), and renal crisis are higher in SSc patients than in the general population and underlie a high mortality rate. No disease-modifying therapy (DMT) has been approved to treat SSc. Systemic immunosuppressants are prescribed to treat ILD and skin fibrosis, while symptoms of PAH and vasculopathy are treated with varying combinations of calcium-channel blockers, endothelin receptor antagonists, PDE-5 inhibitors, and prostacyclin analogues. Renal crisis is treated acutely with ACE inhibitors, which interviewed experts credit with having reduced mortality from this complication. Still, current SSc treatments are only marginally effective, leaving tremendous need for effective DMTs and antifibrotics to treat this debilitating condition. Several marketed drugs (e.g., Boehringer Ingelheim’s Ofev, Roche’s Actemra/RoActemra) are in Phase III studies in SSc.
Niche & Rare Disease Landscape & Forecast: Comprehensive market intelligence providing world-class epidemiology, keen insight into current treatment paradigms, in-depth pipeline assessments, and drug forecasts supported by detailed primary and secondary research.