Spinal Muscular Atrophy Treatment Market is Expected to Reach $3.19 bn by 2027

Spinal Muscular Atrophy is an inherited autosomal recessive neuromuscular disease resulting in the loss of control of skeletal muscle activity such as speaking, walking, breathing, and swallowing, leading to muscle weakness and atrophy.

There are four types of spinal muscular atrophy, namely, SMA type I (Werdnig-Hoffmann disease/ infantile-onset SMA), SMA type II (intermediate form), SMA type III (Kugelberg-Welander disease) and SMA type IV. Causative factors for spinal muscular atrophy include survival motor neuron 1 gene (SMN1) defect on chromosome 5q and mutations on VAPB gene on chromosome 20, DYNC1H1 gene on chromosome 14, BICD2 gene on chromosome 9, and UBA1 gene on the X chromosome.

There is no complete treatment for spinal muscular atrophy but symptomatic treatment is given to prevent the complications. Posture improvement and muscle weakness can be resolved by adopting physical therapy, occupational therapy, and rehabilitation along with therapeutics.

According to Spinal Muscular Atrophy (SMA) Foundation, it is estimated that 1 in 6,000 to 10,000 children are born with SMA and 1 in 40 to 50 people (approximately 6 million Americans) are carriers of the SMA gene. Newborn Screening Initiatives and early diagnosis of disease will enhance effective treatment for children with spinal muscular atrophy.

The more established drug for spinal muscular atrophy is spinzara, reported $364 million quarterly revenue and the newer drug Zolgensma reaped $175 million in revenue in the year 2018. Both these drugs hold major market share and impact the market growth positively in the forecast period.

Increased prevalence and rising awareness of spinal muscular atrophy, increase in R&D activities are the key market drivers. Orphan drug designation to under-development products is aiding the spinal muscular atrophy market. Research and Care foundations spreading awareness about spinal muscular atrophy and the development of proper treatment will raise the demand for treatment market. The key market restraints include high treatment cost and a smaller number of approved products.

Commercialization of products in association with collaborative funding might fasten the drug release process in the market. Development of therapeutics with a novel mechanism of action, improved efficacy, accessibility, ease of use, and patient compliance is the major challenge of spinal muscular atrophy market.

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Major companies for the Treatment of Spinal Muscular Atrophy include Novartis AG, F. Hoffmann-La Roche AG, Cytokinetics, Astellas Pharma Inc, Scholar Rock, Inc., Biogen Inc, and many more…. According to clinicaltrials.gov, there are around 58 active interventional trials for the treatment of Spinal Muscular Atrophy at present.

Recent Updates

  • In collaboration with Astellas, Cytokinetics is developing Reldesemtiv (CK-2127107) for the treatment of Spinal Muscular Atrophy

  • Recently FDA granted priority review to Roche’s risdiplam for spinal muscular atrophy

  • The Food and Drug Administration (FDA) has granted Orphan Drug designation to reldesemtiv developed by Cytokinetics

Based on Treatment (Market Size and Forecast, and Y-o-Y Growth, (US$ Mn):

  • Gene Replacement Therapy

  • Drug Therapy

Based on Geographic Region (Market Size and Forecast, and Y-o-Y Growth, (US$ Mn)

  • North America

  • Europe

  • APAC


Research Scope

  • Provides a detailed Analysis of the Market Structure along with forecast of the various segments and sub-segments.

  • Provides a Comparative Analysis of Key Marketed and Pipeline Products.

  • Provides Key Information on Players involved.

  • Provides a Complete Overview of Market Segments and the Regional Outlook.

  • Provides In-depth Coverage of Key News, including Major Mergers, Acquisitions and Product Development updates such as clinical trial progression updates and regulatory updates.

The Report Provides Key Insights on

  • History of the Spinal Muscular Atrophy Treatment Market, 2015 to 2019

  • Forecast of the Spinal Muscular Atrophy Treatment Market Growth till the year 2027

  • The key market drivers, restraints, challenges, future opportunities and the market dynamics driving the Spinal Muscular Atrophy Treatment Market

  • Analysis of potential growth segments which will drive the market

  • Landscape analysis of the major companies, and new market entrants and companies which possess disruptive technologies which can change the trend of the entire market

  • Key market approaches adopted by the organizations and in-depth intelligence of potential strategies which could alter the market dynamics

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