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The Global SpinalMuscular 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 byRegion (North America, Latin America, Europe, Asia Pacific, Middle East, andAfrica) was estimated to be valued at US$ 884 million in 2017 and is projectedto exhibit a CAGR of 12.4% over the forecast period (2018 – 2026). Spinalmuscular atrophy market is expected to grow at significant rate, owing torising number of clinical trials being conducted to develop effective treatmentfor the disease.
Increasing focus on clinicaltrials is expected to drive growth of the spinal muscular atrophy (SMA) market
Increasing number of clinicaltrials are being conducted by key industry players and rising governmentsupport for these clinical trials is expected to drive growth of the spinalmuscular atrophy market. Various clinical studies focus on therapeuticstrategies to increase survival motor neuron (SMN) protein in motor neuronssince these neurons control muscle movement in the body. SMN protein isimportant for maintenance of motor neurons, which are located in the spinalcord and brainstem, the part of the brain that is connected to the spinal cord.
Currently, only one approved drugtreatment is available for spinal muscular atrophy i.e. Spinraza. In 2016, theU.S. FDA approved Spinraza (nusinersen) drug from Biogen for the treatment ofSMA. Spinraza targets the underlying defect in SMA, so it can help prevent, delay,or even reverse the symptoms. However, its common side effects include higherrisk of constipation and respiratory tract infection along with minimal risk ofkidney problems and bleeding.
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Several biotechnology andbiopharmaceutical giants are working towards developing effective treatment forSMA. For instance, in 2017, AveXis received FDA approval for manufacturing ofAVXS-101 gene therapy. It is currently in development stage, and has beengranted Orphan Drug Designation in 2017 for the treatment of all types ofspinal muscular atrophy (SMA) and Breakthrough Therapy Designation in 2016 forthe same. It also received Fast Track Designation in 2016, for the treatment ofSMA Type 1—one of the most chronic neurological genetic disorders.
In April 2018, AveXis announcedlaunching of a Phase 3 trial, for STR1VE in infants with SMA type 1. AveXisalso plans to initiate a similar trial in Europe called STR1VE EU.
Browse 32 Market Data Tables and26 Figures spread through 161 Pages and in-depth TOC on “Spinal MuscularAtrophy Market, by Disease Type (Type I, Type II, Type III, Type IV), byTreatment (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”
Key players are focusing onmergers and acquisitions, new product launches, and partnerships, in order toenhance their share in the market. For instance, in 2016, Ionis discoveredSpinraza 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 ofcontinuing clinical trials for Olesoxime. The company is currently planning aPhase 3 study enrolling individuals with SMA type II or III.
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Key Takeaways of the SpinalMuscular Atrophy Market:
The global spinal muscularatrophy 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 drugssegment is estimated to hold the dominant position in the spinal muscularatrophy market in 2017 due to approval of single drug Spinraza.
Among disease type, the type Isegment is estimated to hold the dominant position in the spinal muscularatrophy market in 2017. This is owing to it been the most common and severeform 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 dominantposition in 2017 owing to higher demand for spinal muscular atrophy treatmentas a result of higher diagnosis rate in the region.
Key players operating in theglobal spinal muscular atrophy market include Biogen, Cytokinetics, Inc., F.Hoffmann-La Roche, Novartis AG, Pfizer, and Ionis Pharmaceuticals, Inc.
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