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Published Date: 2015-10-16Number of Pages: 44

Spinal Muscular Atrophy Market - Pipeline Assessment, Size, Growth, Trends, and Forecast 2015 - 2023

Spinal Muscular Atrophy Market: Pipeline Analysis Snapshot 

The number of people affected with spinal muscular atrophy is increasing globally. As stated in a publication by CureSMA, at least one in fifty people carry the defective spinal muscular atrophy gene. It should be noted that the research does not include the entire global population and therefore the total number and proportion of SMA gene carriers can be much higher than estimated. This is a key reason for the growth of research and development efforts put into understanding spinal muscular atrophy and discovering feasible treatments for it.

The market is, however, currently restrained by the overall lack of research in terms of the types of spinal muscular atrophy that exist. Research organizations and government bodies are constantly working to measure the growth of spinal muscular atrophy in concerned regions. However, they do not include the various types of spinal muscular atrophy in their research, thus creating a large gap in the mapping of SMA.

There is currently no treatment available for spinal muscular atrophy. Most options available only target the symptoms and not the clinical problem. At the same time, the pipeline for spinal muscular atrophy is fairly dry except for the presence of a few advanced drugs.

North America Scores Highest on SMA Treatment Market Attractiveness

Taking several factors including regulation policies, drug pricing, competitive intensity, and population, it can be said that North America leads in most sub-segments. Researchers and manufacturers concerned with the spinal muscular atrophy are therefore looking to the advanced healthcare infrastructure of North America and Europe.

The North America demand for spinal muscular atrophy treatments largely stems from the U.S. This region shows a higher diagnosis rate of spinal muscular atrophy. It also holds a high amount of government incentives that are given for the development of orphan drugs. The high price of spinal muscular atrophy treatment can be mitigated by the availability of feasible reimbursement policies. Additionally, a large portion of the pharmaceutical industry policies are in favor of this market.

Orphan Drugs to Get Greater Government Incentives

With the U.S. and Europe already having distinguished spinal muscular atrophy as an orphan drug, the government incentives for this category are expected to increase accordingly. Governments are trying to encourage research and development organizations for spinal muscular atrophy to expedite the process of drug development and drug discovery.

Most of the pipeline drugs for spinal muscular atrophy are given several economic advantages such as the clinical research tax credit, a priority new drug application review. This applies especially to the pipeline drugs being developed in the U.S. Similar benefits are also being given to SMA drug developers in Europe. In 2015, only one drug had cleared through to the third phase of clinical and can be expected to enter the market in 2018.

The key players included in the spinal muscular atrophy pipeline assessment are AveXis, Inc., Cytokinetics, Inc., F. Hoffmann-La Roche AG, Novartis AG, and Ionis Pharmaceuticals, Inc. The key pipeline drugs for spinal muscular atrophy currently include Olesoxime (TRO19622), LMI070, RO6885247, RG3039, scAAV9.CB.SMN, CK-2127107, and ISIS-SMNRx.

Chapter 1 Introduction 
1.1 Report Description 
1.2 Research Methodology 
1.3 Market Segmentation
1.4 Assumptions

Chapter 2 Executive Summary
2.1 Pipeline Analysis: Spinal Muscular Atrophy- Market Snapshot

Chapter 3 Pipeline Analysis: Spinal Muscular Atrophy Market Overview 
3.1 Market Definition and Overview
3.2 Current Treatment for Spinal Muscular Atrophy 
3.3 Market Dynamics 
      3.3.1 Drivers 
      3.3.2 Restraints 
      3.3.3 Opportunities
3.3 Market Attractiveness Analysis

Chapter 4 : Pipeline Analysis: Spinal Muscular Atrophy 
4.1 Spinal Muscular Atrophy: Pipeline Overview 
4.2 Late Stage Candidates (Phase 3) 
      4.2.1 ISIS-SMN Rx 
4.3 Early Stage Candidates (Phase 1 and Phase 2) 
      4.3.1 Olesoxime (TRO19622)
      4.3.2 LMI070 
      4.3.3 RO6885247
      4.3.4 CK-2127107
      4.3.5 scAAV9.CB.SMN
      4.3.6 RG3039

Chapter 5 Recommendation 

Chapter 6 Company Profiles 
6.1 AveXis, Inc. 
6.2 Cytokinetics, Inc. 
6.3 F. Hoffmann-La Roche Ltd. 
6.4 Novartis AG 
6.5 Isis Pharmaceuticals, Inc.


Pipeline analysis of spinal muscular atrophy (SMA) market report provides with comprehensive analysis of drugs in clinical trials for treatment for spinal muscular atrophy. The report provides insights to SMA prevalence, and current treatment pattern. The report also identifies key market drivers, restraints and opportunities in the global spinal muscular atrophy therapeutics market. Detailed market attractiveness analysis by geography provides future outlook to the spinal muscular atrophy landscape. Market attractiveness analysis considers various factors such as patient population, drug pricing policies, regulatory restrictions, current competition intensity, and current state of healthcare sector for assessing the potential of each geographical market.

In the pipeline analysis of spinal muscular atrophy (SMA) market report, market analysis and forecast has been provided for late stage (phase 3) candidates, while also includes thorough qualitative information for other candidates in early stage (phase 1 and phase 2). ISIS-SMNRx by Isis Pharmaceuticals is presently the only late stage candidate in spinal muscular atrophy pipeline, while three drugs are currently in phase 2 and three others in phase 1. These include Olesoxime (TRO19622), LMI070, RO6885247, CK-2127107, scAAV9.CB.SMN and RG3039. The report also mentions some of the potential preclinical candidates in the spinal muscular atrophy pipeline. The pipeline analysis of spinal muscular atrophy (SMA) market report provides overview of the clinical trials, and its current status for each drug candidate for treatment of spinal muscular atrophy. Details on mergers, acquisitions, licensing agreements, and other collaborative agreements, updated on regulatory policies, and expected study completion are also mentioned in the report. Market estimation and forecast for phase 3 drugs for treatment of spinal muscular atrophy has been provided from the expected year of drug approval, till 2023 (in USD Million) for the United States.

Pipeline analysis of late stage (phase 3) drugs for treatment of spinal muscular atrophy adopts patient based approach, in which actual patient population that can be targeted by the new drug is considered to arrive at probable sales of the drug in the launch year. Forecast model considers various macro- as well as micro-economic factors such as drug efficiency, side effects, prevalence and incidence rate, impact of regulatory policies, impact of competitor drugs and generic competition (if applicable) and pipeline status. The report collates information from various government as well as corporate sources such as Europe and the U.S. clinical trial registries, World Health Organization (WHO), Centers for Disease Control and Prevention (CDC), CureSMA, Families of SMA, PubMed, company press releases, annual reports and investor presentations. The analysis also considers inputs from industry experts and key opinion leaders with expertise in rare disease drug discovery, and marketing and sales experts.  

Key players with potential candidates in clinical trials for treatment of spinal muscular atrophy have been profiled in the report. These include AveXis, Inc., Cytokinetics, Inc., F. Hoffmann-La Roche Ltd., Novartis AG, and Ionis Pharmaceuticals, Inc. Each of the company profile provides details such as company overview, business overview, financial overview, product portfolio, business strategies, and recent developments.

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