Spinal Muscular Atrophy – Landscape & Forecast – Niche & Rare Disease Landscape & Forecast (US/EU5)

Spinal muscular atrophy (SMA) is a neuromuscular disease characterized by progressive muscle weakness and 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 diseasemodifying therapies have been approved for SMA: Biogen’s nusinersen (Spinraza), Novartis / AveXis’s onasemnogene abeparvovec (Zolgensma), and Roche / Genentech / PTC Therapeutics’ risdiplam (Evrysdi). In November 2025, the FDA approved onasemnogene abeparvovecbrve (Itvisma), an intrathecal formulation of Zolgensma, expanding available treatment options. Although these therapies have broadened treatment options, they remain associated with challenges, including high costs that limit access. This report explores the clinical and commercial potential of key current and emerging therapies for SMA.

Questions answered

• How large is the treatable SMA population, and how will its size change through 2035?
• What is the state of treatment of SMA? Which are the most important drugs and why? What are the interviewed experts’ insights on current treatments? What clinical needs remain unfulfilled?
• What pipeline products are most promising, and what sales / uptake could they secure in SMA?
• What are the drivers and constraints in the SMA market, and how will this market evolve over the forecast period?

Content highlights

Geography: United States and EU5

Primary research:

• Country-specific interviews with thought-leading neurologists
• Survey data collected for this and other Clarivate research

Epidemiology: Diagnosed prevalence of spinal muscular atrophy by country, segmented by subtype (I-IV)

Forecast: Drug-level sales and patient share of key spinal muscular atrophy therapies in 2025 and 2035

Drug treatments: Coverage of key current and late-phase emerging therapies

Product description

Niche & Rare Disease Landscape & Forecast offers comprehensive market intelligence with world-class epidemiology, keen insight into current and emerging therapies, and drug forecasts supported by detailed primary and secondary research, enabling you to:

• Optimize your long-term disease and development strategy.
• Quantify market potential for your pipeline assets and those of your competitors.
• Understand a disease from top to bottom, including key patient populations, the current and future therapeutic landscape, and the evolving market trajectory.
• Gauge the commercial outlook and impact of key market events.