The Global Spinal Muscular Atrophy (SMA) Market, by Disease Type (Type I, Type II, Type III, Type IV) Treatment (Gene Therapy and Drugs), by Age (Infant and Adult),and by Region (North America, Latin America, Europe, Asia Pacific, Middle East, and Africa) was estimated to be valued at US$ 884 million in 2017 and is projected to exhibit a CAGR of 12.4% over the forecast period (2018 – 2026). Spinal muscular atrophy market is expected to grow at significant rate, owing to rising number of clinical trials being conducted to develop effective treatment for the disease.

Increasing focus on clinical trials is expected to drive growth of the spinal muscular atrophy (SMA) market

Increasing number of clinical trials are being conducted by key industry players and rising government support for these clinical trials is expected to drive growth of the spinal muscular atrophy market. Various clinical studies focus on therapeutic strategies to increase survival motor neuron (SMN) protein in motor neurons since these neurons control muscle movement in the body. SMN protein is important for maintenance of motor neurons, which are located in the spinal cord and brainstem, the part of the brain that is connected to the spinal cord.

Currently, only one approved drug treatment is available for spinal muscular atrophy i.e. Spinraza. In 2016, the U.S. FDA approved Spinraza (nusinersen) drug from Biogen for the treatment of SMA. Spinraza targets the underlying defect in SMA, so it can help prevent, delay, or even reverse the symptoms. However, its common side effects include higher risk of constipation and respiratory tract infection along with minimal risk of kidney problems and bleeding.

Several biotechnology and biopharmaceutical giants are working towards developing effective treatment for SMA. For instance, in 2017, AveXis received FDA approval for manufacturing of AVXS-101 gene therapy. It is currently in development stage, and has been granted Orphan Drug Designation in 2017 for the treatment of all types of spinal muscular atrophy (SMA) and Breakthrough Therapy Designation in 2016 for the same. It also received Fast Track Designation in 2016, for the treatment of SMA Type 1—one of the most chronic neurological genetic disorders.

In April 2018, AveXis announced launching of a Phase 3 trial, for STR1VE in infants with SMA type 1. AveXis also plans to initiate a similar trial in Europe called STR1VE EU.

Browse 32 Market Data Tables and 26 Figures spread through 161 Pages and in-depth TOC on “Spinal Muscular Atrophy Market, by Disease Type (Type I, Type II, Type III, Type IV), by Treatment (Gene Therapy and Drugs), by Age (Infant and Adult), and by Region (North America, Latin America, Europe, Asia Pacific, Middle East, and Africa) - Global Forecast to 2026”

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Key players are focusing on mergers and acquisitions, new product launches, and partnerships, in order to enhance their share in the market. For instance, in 2016, Ionis discovered Spinraza and co-developed the drug with Biogen. Ionis received a payment of US$ 320 million from Biogen for the development of Spinraza till the year 2016, including US$ 90 million based on regulatory approvals in Japan and Europe. Another key player Roche, acquired Trophos in 2015 with the intent of continuing clinical trials for Olesoxime. The company is currently planning a Phase 3 study enrolling individuals with SMA type II or III.

Key Takeaways of the Spinal Muscular Atrophy Market:

  • The global spinal muscular atrophy market is expected to exhibit a CAGR of 12.4% over the forecast period, owing to rising number of clinical trials for development of new therapies.
  • Among treatment, the drugs segment is estimated to hold the dominant position in the spinal muscular atrophy market in 2017 due to approval of single drug Spinraza.
  • Among disease type, the type I segment is estimated to hold the dominant position in the spinal muscular atrophy market in 2017. This is owing to it been the most common and severe form of SMA. According to SMA Europe 2015 data, type I accounts for 50% to 70% of cases of childhood onset SMA.
  • North America held a dominant position in 2017 owing to higher demand for spinal muscular atrophy treatment as a result of higher diagnosis rate in the region.
  • Key players operating in the global spinal muscular atrophy market include Biogen, Cytokinetics, Inc., F. Hoffmann-La Roche, Novartis AG, Pfizer, and Ionis Pharmaceuticals, Inc.
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