Spinal muscular atrophy (SMA) is a neuromuscular disease characterized by progressive muscle weakness; it is a leading genetic cause of death in infants. SMA is divided into four clinical subtypes based on severity and age at symptom onset. Since 2016, three disease-modifying therapies have been approved for SMA: Biogen’s Spinraza (nusinersen), Novartis / AveXis’s Zolgensma (formerly AVXS-101; onasemnogene abeparvovec), and Roche / Genentech / PTC Therapeutics’ Evrysdi (risdiplam). Although these therapies have expanded treatment options for patients, the therapies are associated with many challenges, including high prices, which limit patients’ access to them. In the SMA pipeline are many therapies with diverse mechanisms of action and routes of administration; if approved, they will allow physicians to treat patients based on their symptoms, comorbidities, and preferences. In this content, we will explore the clinical and commercial potential of key current and emerging therapies for SMA.
Geography: United States and EU5.
Primary research: Six country-specific interviews with thought-leading neurologists supported by survey data collected for this study.
Epidemiology: Diagnosed prevalence of SMA by country, segmented by subtype (I-IV).
Forecast: Drug-level sales and patient share of key SMA therapies through 2031.
Emerging therapies: Phase III/PR: 3drugs; Phase II: 1drug; coverage of select early-phase products
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.
This Niche & Rare Disease Landscape & Forecast report 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.