Spinal Muscular Atrophy Market– A Genetic Disorder Affecting Part of the Nervous System
Spinal muscular atrophy (SMA) is a genetic disorder that affects the control of muscle movement. It is caused due to loss of specialized nerve cells, called motor neurons, in the spinal cord and brainstem. The loss of motor neurons leads to weakness and atrophy of muscles used for activities such as sitting up, walking, crawling, and controlling head movement. In severe cases, the muscles used for swallowing and breathing are also affected. There are four types of spinal muscular atrophy, which are differentiated based on the severity of muscle weakness, pattern of features, and age, when the muscle problems begin. These are classified as type 1, 2, and 3 that appears in childhood. Type 4 appears in adulthood and is also termed as adult onset SMA.
The global spinal muscular atrophy market size was valued at US$ 884 million in 2017 and is expected to witness a robust CAGR of 12.4% over the forecast period (2018–2026).
Figure 1. Global Spinal Muscular Atrophy Market Share (%), By Age
Source: Coherent Market Insights Analysis (2018)
Increasing number of clinical trials and government funding for R&D is expected to bolster the spinal muscular atrophy market growth
Various government and private organizations are investing huge capital and resources towards the development of new and effective therapies for spinal muscular atrophy. For instance, in 2015, the SMA Foundation financed over US$ 60 Mn in drug development programs and essential drug discovery assets that aim to develop an effective SMA therapy. In 2013, Cure SMA granted fund to Cytokinetics for the study of Tirasemtiv for SMA. Financial details of the grant were not disclosed. Tirasemtiv is a novel skeletal muscle activator and a lead drug candidate from Cytokinetics' skeletal muscle contractility program. It has been studied in clinical trials for individuals with amyotrophic lateral sclerosis (ALS). Similar to SMA, ALS also affects motor neurons.
The National Institute of Neurological Disorders and Stroke (NINDS), a part of the National Institutes of Health (NIH), conducts clinical, translational, and basic research on SMA and also supports research through grants to major medical institutions across the U.S. In 2011, the authority established NeuroNext clinical trials network to promote rapid development and implementation of trials for neurological disorders that affect adults and/or children. The network is designed to develop early-phase trials aimed at identifying biomarkers that can be measured to determine the presence and severity of a disease—and testing promising, emerging therapies.
Figure 2. Spinal Muscular Atrophy Therapy Pipeline, 2017
Increasing incidence rate of spinal muscular atrophy worldwide is expected to augment the market growth
Spinal muscular atrophy mainly manifests early in life and therefore is a major genetic cause of death in infants. It is one of the most common rare diseases. According to SMA Foundation 2015 statistics, spinal muscular atrophy has generally been believed to affect as many as 10,000 to 25,000 adults and children in the U.S. In March 2018, CureSMA stated that SMA affects around 1 in 11,000 children, and 1 in 50 is a carrier of the disease. It has been found in people of every race, however, is most common in Caucasians, of whom 1 in 35 is a carrier. Spinal muscular atrophy carrier rates for other populations include Asians (1 in 53), African Americans (1 in 66), and Hispanics (1 in 117).
However, high cost of therapy can confine growth of the spinal muscular atrophy market during the forecast period. For instance, Biogen’s Spinraza (nusinersen) drug for spinal muscular atrophy is estimated to cost US$ 125,000 per injection, accounting for US$ 750,000 for the first year and US$ 375,000 later.
Major players operating in the global spinal muscular atrophy market include Biogen, Cytokinetics, Inc., F. Hoffmann-La Roche, Novartis AG, Pfizer Inc., and Ionis Pharmaceuticals, Inc.