Spinal Muscular Atrophy | Landscape & Forecast | Disease Landscape & Forecast

Publish date: September 2018

Login to access report

Spinal muscular atrophy (SMA) is an autosomal recessive disease characterized by progressive muscle weakness due to the loss of anterior horn motor neurons. Nearly all cases of SMA are caused by homozygous deletion of mutation of the SMN1 gene. SMA ranges widely in severity, and symptom onset usually occurs during childhood. In its most serious form, SMA is lethal in infancy. Until recently, no treatments existed for SMA. Biogen/Ionis’s antisense therapy Spinraza now offers a targeted treatment option for SMA patients, and several exciting pharmacological (e.g., Roche’s RG-7916) and gene (Novartis/AveXis’s AVXS-101) therapies are in late-stage development, seeking to capitalize on pressing unmet treatment need in this devastating condition.

Questions Answered

  • How large is the treatable SMA population and how will its size change between 2017 and 2027?
  • What is the current state of treatment in spinal muscular atrophy? How many patients within each disease subtype are receiving Spinraza?
  • What clinical needs remain unfulfilled?
  • What pipeline products are viewed by spinal muscular atrophy treatment providers as most promising, and what sales/uptake will they achieve in spinal muscular atrophy? Which notable early-stage therapies are progressing through development?
  • What are the drivers and constraints in the spinal muscular atrophy market, and how will the market evolve over the forecast period?

Product Description

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.

Table of contents

  • Disease Landscape & Forecast
    • Commercial Outlook
      • Market Outlook
        • Key Findings
      • 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
        • Troponin-Tropomyosin Complex Activators
        • SMN1 Corrective Therapies
    • Forecast
    • Etiology and Pathophysiology
      • Disease Overview
      • Etiology
        • Homozygous Deletion of the SMN1 Gene
        • Non-5q SMAs
        • The SMN2 Gene: A Modifier of Disease Course and Severity
      • Pathophysiology
        • The SMN Protein
        • Pathophysiological Processes in Spinal Muscular Atrophy
      • Spinal Muscular Atrophy Clinical Subtypes
      • Spinal Muscular Atrophy Clinical Presentation
      • Key Pathways and Drug Targets
    • Epidemiology
      • Key Findings
      • Epidemiology Populations
        • Diagnosed Prevalent Cases by Spinal Muscular Atrophy Type
        • Drug-Treated Cases of Spinal Muscular Atrophy
    • Current Treatment
      • Key Findings
      • Diagnosis
        • Diagnosis
        • Diagnostic Tests
        • Treatment Providers and Referral Patterns
      • Treatment Goals
      • Key Current Therapies
        • Overview
        • SMN2-Splicing Modulators
        • Short-Acting Beta2 Agonists
      • Medical Practice
        • Overview
        • Spinraza
        • Pulmonary Care
        • Nutritional Support
        • Orthopedic Care
        • Region-Specific Treatment Practices
    • Unmet Need Overview
      • Current and Future Attainment of Unmet Needs in Spinal Muscular Atrophy
    • Emerging Therapies
      • Introduction
        • Key Findings
      • Key Emerging Therapies
        • Notable Developments Among Key Emerging Therapies for Spinal Muscular Atrophy
        • SMN2-Splicing Modulators
        • Troponin Tropomyosin Complex Activators
        • SMN1 Corrective Therapies
      • Early-Phase Pipeline Analysis
        • Notable Developments in the Early-Phase Pipeline for Spinal Muscular Atrophy
      • Key Discontinuations and Failures in Spinal Muscular Atrophy
      • Patient Registries
        • Patient Organizations
      • Orphan Drug Designation
        • Orphan Drug Provisions: United States
        • Orphan Drug Provisions: Europe
    • Access and Reimbursement Overview
      • Looking for More?
      • Region-Specific Reimbursement Practices
        • United States
        • EU5
    • Methodology
      • Bottom-Up Forecasting Overview
        • Patient Populations
        • Drug-Specific Assumptions
      • Bottom-Up Forecast Assumptions
        • General Sources of Data
        • General Statements About Pricing
        • Dosing, Days of Therapy, and Compliance
        • Out-Year Forecasting
        • Emerging Therapy Prices
      • Primary Market Research
        • Experts Interviewed
    • Appendix
      • Spinal Muscular Atrophy Bibliography

Author(s): Ian Love, PhD; Johnson Olabisi, MBBS, MSc

Ian is an analyst on the Infectious, Niche, and Rare Diseases team at Decision Resources Group, specializing in niche and rare diseases. Ian conducts primary and secondary research to gauge the market landscapes of these diseases, thoroughly analyzing disease etiology, current treatments, unmet need, and emerging therapies expected to impact their respective markets. He received his doctorate in biomedical sciences from the University of Massachusetts Medical School and his in cell & molecular biology from the Worcester Polytechnic Institute. Prior to joining Decision Resources Group, Dr. Love was an Instructor in the Department of Internal Medicine at Virginia Commonwealth University.

Johnson joined Decision Resources Group (DRG) in 2015 and with the Epidemiology team develops epidemiological populations forecasts for different infectious and non-communicable diseases with his particular interests in the oncology space. Prior to joining DRG, he trained as a community physician where he was involved in primary (patient) care, primary health care and various community research & activities. He has also supervised and coordinated various governmental and non-governmental public health projects. Johnson holds a Masters of Science in Public Health (Health Economics) degree from the London School of Hygiene and Tropical Medicine with a Masters in Epidemiology and Medical Statistics & a Medical degree from the University of Ibadan, Nigeria.