+1-518-618-1030 866 - 552 - 3453 USA-Canada Toll Free
Published Date: 2015-10-16Number of Pages: 44

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


Spinal muscular atrophy (SMA) is a hereditary autosomal recessive disease affecting areas of the nervous system that control voluntary muscle movement. Mutation in survival motor neuron (SMN) gene causes deficiency of SMN protein. This results in motor neurons to lose control over voluntary muscle movement. SMA is a leading fatal genetic disease among infants; where one infant in 6,000 births is estimated to suffer from SMA. Furthermore, over 10 million people in the U.S. are carriers of SMA gene. 

Based on the age of onset and symptoms, spinal muscular atrophy has been classified in four main types: type 1, type 2, type 3, and type 4, and thus the respective markets. Each of these types has a characteristic disease progression and defined mortality rate. Type 1 SMA is the most severe form, and affects infants aged between zero and six months. Approximately 65% of these cases are of type 1 SMA, wherein infants usually do not survive for more than 2 years. Type 2 SMA is a moderate form affecting children aged between six months and eighteen months. Type 3 and 4 are less severe and usually have a normal lifespan. Spinal muscular atrophy is diagnosed by identifying mutation in survival of motor neuron (SMN) gene. Other diagnostic tests include muscle biopsy, electromyogram (EMG), and nerve conduction velocity.

Currently, no cure exists for spinal muscular atrophy, and the treatment is targeted towards symptoms management. Physiotherapy is strongly recommended in early stages of the disease to extend range of motion and improve muscle flexibility. Spinal muscular atrophy drugs pipeline is dry; however, it has three drugs in phase 1, three in phase 2, and one in phase 3 clinical trials. ISIS-SMNRx, an antisense therapy candidate by Isis Pharmaceuticals, is currently under phase 3 studies for treatment of type 1 and 2 SMA. If approved, ISIS-SMNRx would be the only drug available of the treatment of spinal muscular atrophy, creating lucrative opportunity for Isis Pharmaceuticals and Biogen Idec. Phase 3 study is expected to complete in June 2017. The drug is projected to record sales of USD 468.2 million in the U.S. by 2023.

Olesoxime (TRO19622) has completed its phase2/3 trials, and has shown promising results, with potential to effectively treat type 2 and type 3 SMA. Other potential candidates include Olesoxime (TRO19622), LMI070, RO6885247, CK-2127107, scAAV9.CB.SMN and RG3039. Key players with potential therapeutics in clinical trial phase include AveXis, Inc., Cytokinetics, Inc., F. Hoffmann-La Roche Ltd., Novartis AG, and Isis Pharmaceuticals, Inc. 



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.