Spinal Muscular Atrophy (SMA) is a neuromuscular disorder possessing an autosomal recessive heredity genetic profile. SMA is one of the fatal diseases with an incidence rate of 1 per 6,000 birth to 1 per 10,000 births. The heterozygote carrier frequency of SMA is about 1 in every 40 to 60 people across the globe. It severely affects the motor neurons in the brain and spinal cord due to the homozygous mutation of the survival motor neuron gene 1 (SMN1). These motor neurons bring about the transfer of chemical and electrical charge to and fro from the voluntary muscles, present all over the body. Thereby, the affected motor neurons in the brain and spinal cord causes difficulty in performing several physical activities, including swallowing, crawling, and walking. SMA is a difficult disorder to diagnose, depending upon both histological and electrophysiological evidence of muscle denervation with uncertain results of treatment.
The global spinal muscular atrophy treatment market size was valued at USD 948.7 million in 2018.
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Following Are the Clinical Features of the Spinal Muscular Atrophy Disease
Globally, the rising prevalence of SMA can be attributed to the carrier frequency of defective DNA with a deleted SMN1 gene, the unavailability of treatment options, and lack of awareness. However, rapid technological advancements, rising awareness, reimbursement scenarios, and government initiatives are driving the spinal muscular atrophy treatment market. The treatment options for SMA are limited, and so is the adoption rate across the Asia Pacific, Latin America, and the Middle East, which is further hampering the market growth to some extent. However, the presence of a plethora of drugs and other treatment options in the clinical pipeline is expected to act as the backbone for market growth.
Global spinal muscular atrophy treatment market is segmented into SMA type, treatment, route of administration, and region.
By SMA type, the global market is segmented into severe (type I, severe SMA or WerdnigHoffmann disease), intermediate (type II or chronic SMA), mild (type III, juvenile SMA or Kugelberg-Welander disease), and type IV (adult SMA). Severe or type I SMA is the most common type of spinal muscular atrophy disease and affects the children aged up to 6 months. The patient loses the ability to sit, and the life expectancy is less than 2 years. Rising prevalence of Type I SMA, ongoing R&D activities, and a large number of drugs in the clinical pipeline stage is expected to drive the segment growth. Globally, about half of the SMA patients suffer from Type I, which is subsequently increasing the demand for treatment therapies, such as gene therapy. In June 2019, Zolgensma/AVXS-101 developed by Avexis/Novartis was approved by the FDA for patients less than 2 years of age. Similarly, Branaplam, developed by Novartis, is in phase II clinical trial for the treatment of Type I SMA.
Intermediate SMA or Type II affects the patients aged around 6 to 18 months. Out of all the patients with type II SMA, around 70% do not have deep tendon reflexes, which has increased the demand for technologically advanced treatment options and specialized medical equipment. In June 2019, Reldesemtiv by Cytokinetics/Astellas was in phase II clinical trial. Currently, SRK-015 (Topaz) by Scholar Rock is also in phase II clinical trial.
By treatment, the global spinal muscular atrophy treatment market is segmented into gene therapy and drugs. The gene therapy segment is grabbing the highest share in the spinal muscular atrophy treatment market. The treatment is slightly costlier but more effective, compared to other drug treatment options that cost over USD 750,000 every year. In May 2019, Novartis received approval for Zolgensma, gene therapy for the treatment of spinal muscular atrophy, and is expected to cost around USD 2.1 million per person.
Regionally, the global spinal muscular atrophy treatment market is segmented into North America, Europe, Asia Pacific, and Latin America and the Middle East & Africa.
North America: Booming Market for SMA Treatment
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North America is dominating the global spinal muscular atrophy treatment market and will continue the same during the forecast period. This can be attributed to the increasing awareness regarding the disease, rising government initiatives and attractive reimbursement policies, robust healthcare infrastructure, and increasing expenditure power. The region is also backed with the presence of leading players and insurance companies that are actively working to aid the people suffering from spinal muscular atrophy. For instance, Biogen offers reimbursement for the treatment of spinal muscular atrophy using Spinraza, an FDA approved drug, which costs around USD 4 million. SMA 360o by Biogen is a program that offers individualized and comprehensive support to the patients and their families. It also offers education, logistical assistance, financial assistance, and insurance benefits investigation under the program. Similarly, in the region, the government has established a Rare Disease Reimbursement Manager (RDRM) program for providing reimbursement for the Spinraza treatment.
Clinical Pipeline Drugs to Cater to the European Market Growth
Europe is expected to experience significant growth in the spinal muscular atrophy treatment market on account of the increasing number of treatments, drugs, and gene therapies in the clinical pipeline. Spinraza, an SMA treatment drug by Biogen, was approved and marketed in 2016. Similarly, Zolgensma, gene therapy by Avexis, was approved in May 2019. The cost of gene therapy is relatively high to be affordable for low to middle-income families. To attend to the situation, the government is offering umpteen reimbursement policies to the patients and is also working to reduce the cost of gene therapy drugs.
European SMA Drug Pipeline According to the Treatment Target—October 2019
Asia Pacific: Revolutionizing the SMA Disease Management
Spinal muscular atrophy treatment market in India, China, South Korea, Australia, and Japan, is expected to experience significant growth during the forecast period. Cumulatively, the market in these 5 countries is expected to reach USD 2,959.6 million in 2029 from USD 309.4 million in 2018. This can be attributed to the rising approval activities from the governments. In June 2017, Japan was the first to approve Spinraza for the treatment of spinal muscular atrophy. In line with this, South Korea, China, and Australia also approved Spinraza for the treatment as it has a promising impact depicting meaningful improvements in motor functions and increasing survival rare in children. Similarly, the launch of Roche’s Risdiplam, an SMN2 splicing modifier, is expected to boost market growth to some extent.
Symptomatic Treatment: Treating and caring for the people suffering from SMA requires the interdisciplinary management of nutritional and gastroenterological, psychosocial, orthopedic, and respiratory issues. The availability of regional sources, cultural perspectives, and socioeconomic factors are some of the factors affecting and influencing the implementation of the standard of care. The advances and improvements in care have resulted in publishing an updated version of recommendations on diagnosing SMA based on its clinical features.
Drug Treatment: Investigation of several different compounds in randomized clinical trials, including gene enhancement, gene replacement, and muscle enhancement, has resulted in yielding the key information about the feasibility of patient recruitment and trial designs and outcome measures. Based on the clinical trials and studies, the therapeutic developments can be subdivided into therapies aiming to modify replacing SMN1 gene, upregulating muscle growth, and splicing SMN2. These developments are expected to cater to the growth of the global spinal muscular atrophy treatment market in the years to come.
Emerging New-Born Screening: In the last few years, new-born screening has paved its way to becoming an essential and widely accepted way of diagnosing any genetic disorders or autoimmune diseases in newborns. Through newborn screening, SMA Type I can be diagnosed at the pre-symptomatic stage. It is performed using quantitative polymerase chain reaction (qPCR) assays, which detects homozygous deletions in intron or exon of SMN1 with the help of dried blood spot analysis. New-born screening is expected to drive the market to new heights and is projected to be a useful diagnostic tool for the diagnosis of Type I SMA. In July 2018, spinal muscular atrophy was added to the Recommended Uniform Screening Panel (RUSP) and was also being implemented across the U.S.
List of key spinal muscular atrophy treatment market companies
|Market Size||USD in Billion By 2030|
|Forecast Units||Value (USD Million)|
|Report Coverage||Revenue Forecast, Competitive Landscape, Growth Factors, Environment & Regulatory Landscape and Trends|
|Segments Covered||by SMA Type (Severe, Mild), Treatment (Gene Therapy, Drugs), Route of Administration (Oral, Intrathecal)|
|Geographies Covered||North America, Europe, Asia-Pacific, LAME and Rest of the World|
|Key Companies Profiled/Vendors||Pfizer Inc., Biogen Idec, Boehringer Ingelheim GmbH, Ionis Pharmaceuticals, Inc., Avexis, Inc., Novartis AG, Cytokinetics, Inc., FHoffmann-La Roche AG, and Regeneron Pharmaceuticals, Inc.|
|Key Market Opportunities||High Demand In Healthcare It Sector Is Pushing Spinal Muscular Atrophy Treatment Market|