Spinal Muscular Atrophy Market: Phase 3 Candidate ISIS-SMN Rx Shows Promising Results in Clinical Trial: Transparency Market Research

ALBANY, New York, March 15, 2016 /PRNewswire/ --

Transparency Market Research, in its latest report titled "Spinal Muscular Atrophy Market - Pipeline Assessment, Size, Growth, Trends, and Forecast 2015 - 2023", offers readers a detailed analysis of the pipeline of the global spinal muscular atrophy (SMA) market, in addition to a comprehensive evaluation of the various drugs in different phases of clinical trials for the treatment of spinal muscular atrophy.  

According to report, the spinal muscular atrophy market phase three candidate ISIS-SMNRx is expected to generate total revenue of USD 0.47 billion by 2023.

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Spinal muscular atrophy is genetic neuromuscular disease that is characterized by muscle weakness and atrophy. This disease is the leading genetic cause of death in toddlers and infants, given that it generally manifests early in life. According to the Spinal Muscular Atrophy Association, this disease affects an estimated 10,000 to 25,000 adults and children in the U.S. alone, making it one of the most common rare diseases. The association reveals that an estimated 6 million Americans are carriers of the SMA gene and one in 6,000 to one in 10,000 kids are born with this neuromuscular disease.

The research report offers key insights into the prevalence and current treatment pattern of spinal muscular atrophy, identifying major market drivers, opportunities, and restraints in the global market for spinal muscular atrophy therapeutics. An analysis of market attractiveness with respect to geography takes into consideration several factors such as drug pricing policies, current competition intensity, patient population, regulatory restrictions, and present state of the healthcare sector. This gives readers a clear picture of the potential of each geographical segment of the spinal muscular atrophy market.

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The spinal muscular atrophy therapeutics pipeline is segmented into early stage candidates and late stage candidates. Early stage candidates of Phase 1 and Phase 2 include LMI070, CK-2127107, RG3039, Olesoxime (TRO19622), RO6885247, and scAAV9.CB.SMN. Late stage candidates of Phase 3 include ISIS-SMN Rx. This Phase 3 candidate for the treatment of Type 1 and Type 2 SMA is present an annual sales of US$0.4 bn in the US by the end of the forecast period in 2023.

Europe and the US are presently the dominating markets for spinal muscular atrophy therapeutics owing to the fact that these regions have a greater percentage of diagnosed cases of SMA. Private and government organizations have been largely involved in creating awareness about spinal muscular atrophy. In addition, the governments in these regions have been offering incentives for the development of therapeutic options for the treatment of rare diseases, which in turn boosts research and development in the field of spinal muscular atrophy.

The major companies competing in this space are F. Hoffmann-La Roche Ltd., Isis Pharmaceuticals, Inc., AveXis, Inc., Cytokinetics, Inc., and Novartis AG, and these players have been profiled in the report with the help of several market intelligence tools. Their strengths, weaknesses, opportunities, and threats have been evaluated, along with a detailed assessment of their product offerings.

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Spinal Muscular Atrophy Pipeline Segmentation 

• Late Stage Candidates (Phase 3)
  • ISIS-SMN Rx
• Early Stage Candidates (Phase 1 and Phase 2)
  • Olesoxime (TRO19622)
  • LMI070
  • RO6885247
  • CK-2127107
  • SMN
  • RG3039

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