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.
Key Market Access Considerations in Spinal Muscular Atrophy: United States
General Reimbursement Environment: United States
Key Market Access Considerations in Spinal Muscular Atrophy: EU5
General Reimbursement Environment: EU5
Appendix
Key Abbreviations Related to Spinal Muscular Atrophy
Brands, Marketers, and Generic Availability of Key Therapies for Spinal Muscular Atrophy by Market
Spinal Muscular Atrophy Bibliography
Ian Love
Ian Love, Ph.D., is an analyst on the Infectious, Niche, and Rare Diseases team at DRG, part of Clarivate. Prior to joining DRG, he was an instructor in the Department of Internal Medicine at Virginia Commonwealth University. He received his doctorate in biomedical sciences from the University of Massachusetts Medical School and his B.S. in cell and molecular biology from Worcester Polytechnic Institute.
Johnson Olabisi, M.B.B.S., M.Sc.
Johnson Olabisi, M.B.B.S., M.Sc., is an epidemiologist at DRG, part of Clarivate. Prior to joining DRG, he trained as a community physician. He has also supervised and coordinated various governmental and nongovernmental public health projects. He holds a master’s degree in public health (health economics) from the London School of Hygiene and Tropical Medicine, a master’s degree in epidemiology and medical statistics, and a medical degree from the University of Ibadan in Nigeria.