Spinal Muscular Atrophy – Landscape & Forecast – Disease Landscape & Forecast
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.
Questions answered
- How large is the treatable SMA population, and how will its size change through 2031?
- What is the current state of treatment of SMA? Which are the most important drugs and why? What are 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 therapies of note are progressing in earlier phases?
- 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: 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
Product description
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.
Table of contents
- Spinal Muscular Atrophy - Landscape & Forecast - Disease Landscape & Forecast
- Key findings
- SMA Key Findings - November 2022
- Commercial outlook
- Key findings
- Regional sales of key therapies to treat spinal muscular atrophy: 2021 and 2031
- Spinal muscular atrophy SWOT analysis
- Drivers and constraints
- What factors are driving sales in spinal muscular atrophy?
- What factors are constraining sales in spinal muscular atrophy?
- Drug-class-specific trends
- SMN2-splicing modulators
- SMN1 gene replacement therapies
- Myostatin-inhibitors
- Key findings
- Forecast
- Sales of key therapies in spinal muscular atrophy
- Etiology and pathophysiology
- Disease overview
- Etiology
- Homozygous deletion of the SMN1 gene
- Mutations in SMN1
- Non-5q SMAs
- Genes implicated in non-5q SMAs
- The SMN2 gene: a modifier of disease course and severity
- Pathophysiology
- Critical role of SMN in anterior horn motor neurons
- The SMN protein
- Pathophysiological processes in spinal muscular atrophy
- Clinical subtypes of spinal muscular atrophy
- Clinical presentation of spinal muscular atrophy
- Key signs and symptoms of spinal muscular atrophy
- Key pathways and drug targets
- Disease overview
- Epidemiology
- Key findings
- Epidemiology populations
- Disease definition
- Methods
- Sources used for the diagnosed prevalence of spinal muscular atrophy
- Diagnosed prevalent cases of spinal muscular atrophy: 2021-2031
- Disease definition
- Methods
- Sources used for the prevalence of spinal muscular atrophy by type
- Diagnosed prevalent cases of spinal muscular atrophy in the major pharmaceutical markets by type: 2021-2031
- Drug-treated, diagnosed prevalent cases of spinal muscular atrophy: 2021-2031
- Key findings
- Current treatment
- Key findings
- Diagnosis
- Diagnostic tests
- Treatment providers and referral patterns
- Treatment goals
- Key endpoints used in clinical trials for spinal muscular atrophy
- Key current therapies
- Overview
- Mechanism of action of key drug classes used for spinal muscular atrophy
- Current treatments used for spinal muscular atrophy
- Spinraza
- Clinical trial outcomes for Spinraza
- Advantages and disadvantages of Spinraza
- Key results from select clinical trials investigating Spinraza for the treatment of spinal muscular atrophy
- Key ongoing clinical trials of Spinraza in the treatment of spinal muscular atrophy
- Expert insight: Spinraza
- Evrysdi
- Clinical trial outcomes for Evrysdi
- Advantages and disadvantages of Evrysdi
- Key results from select clinical trials investigating Evrysdi for the treatment of spinal muscular atrophy
- Key ongoing clinical trials of Evrysdi in the treatment of spinal muscular atrophy
- Expert insight: Evrysdi
- Zolgensma
- Clinical trial outcomes in Zolgensma
- Advantages and disadvantages of Zolgensma
- Key results from select clinical trials investigating Zolgensma for the treatment of spinal muscular atrophy
- Key ongoing clinical trials of Zolgensma in the treatment of spinal muscular atrophy
- Expert insight: Zolgensma
- Clinical trial outcomes for albuterol
- Advantages and disadvantages of albuterol
- Key results from select clinical trials investigating albuterol for the treatment of spinal muscular atrophy
- Expert insight: albuterol
- Medical practice
- Overview
- Pulmonary care
- Nutritional support
- Orthopedic care
- Spinal muscular atrophy treatment guidelines by market
- Generalized treatment decision tree for spinal muscular atrophy
- Key findings
- Unmet need overview
- Current and future attainment of unmet needs in spinal muscular atrophy
- Top unmet needs in spinal muscular atrophy: current and future attainment
- Expert insight: unmet need in spinal muscular atrophy
- Drug pipeline
- Pipeline
- Regulatory Milestones
- Indication Comparison
- Emerging therapies
- Key findings
- Pipeline trends for spinal muscular atrophy
- Key emerging therapies
- Key therapies in development for spinal muscular atrophy
- Estimated launch dates of key emerging therapies for the treatment of spinal muscular atrophy
- Reldesemtiv profile
- Analysis of the clinical development program for reldesemtiv
- Expectations for launch and sales opportunity of reldesemtiv in spinal muscular atrophy
- Apitegromab
- Apitegromab profile
- Key ongoing clinical trials of apitegromab in the treatment of spinal muscular atrophy
- Analysis of the clinical development program of apitegromab
- Expert insight: apitegromab
- Expectations for launch and sales opportunities of apitegromab
- Taldefgrobep alfa
- Taldefgrobep alfa profile
- Key ongoing clinical trials of taldefgrobep alfa in the treatment of spinal muscular atrophy
- Analysis of the clinical development program of taldefgrobep alfa
- Expert insight: taldefgrobep alfa
- Expectations for launch and sales opportunities of taldefgrobep alfa
- RG6237
- RG6237 profile
- Key ongoing clinical trials of RG6237 in the treatment of spinal muscular atrophy
- Analysis of the clinical development program of RG6237
- Expectations for launch and sales opportunities of RG6237
- Early-phase pipeline analysis
- Select compounds in early-phase development for spinal muscular atrophy
- Patient registries
- Patient registries for spinal muscular atrophy
- Prominent spinal muscular atrophy patient organizations
- Orphan drug designation
- Key findings
- Access and reimbursement overview
- Region-specific reimbursement practices
- General reimbursement environment: United States
- General reimbursement environment: EU5
- Region-specific reimbursement practices
- Appendix
- Key abbreviations related to spinal muscular atrophy
- Spinal muscular atrophy bibliography
- Key findings
Ansuman Swain, B.D.S., M.P.H., is an associate epidemiologist at Clarivate. As part of his M.P.H. curriculum, he worked as an intern at the National Health Mission in Odisha, India, where he observed the implementation of the RBSK program. Mr. Swain is a graduate in dentistry from KIIT University in Odisha and holds an M.P.H. degree from Manipal Academy of Higher Education in Karnataka, India, with a specialization in global health from Maastricht University in the Netherlands.