Spinal Muscular Atrophy (SMA) Market: Phase 3 Candidate ISIS-SMNRx to Reach US$0.47 bn by 2023

Spinal muscular atrophy is primarily a hereditary autosomal recessive disease that affects the nervous system areas that controls voluntary muscular movement. SMA is commonly identified as a genetic disorder causing mortality among people, especially infants. As per Orphanet, 1 infant out of 6,000 births is predicted to suffer spinal muscular atrophy. More than 10 mn individuals in the United States are carriers of spinal muscular atrophy. The spinal muscular atrophy market phase 3 candidate ISIS-SMNRx is predicted to reach US$0.47 bn by 2023.

Increasing Number of Infants with SMA to Drive Global Market

Recently, as per a news report, a few families in Morgantown found hope for their children who were suffering from spinal muscular atrophy. The families were denied access to the clinical trial drug SMNRx from ISIS Pharmaceuticals. Since then, the families, with the support of many others, have been trying to sign a petition to allow SMA-diagnosed children to have clear access to the relevant drugs for treatment. 

Such and other events have occurred as the result of the growing prevalence of SMA among children. This will propel the global spinal muscular atrophy market in the coming years.


Type 1 Spinal Muscular Atrophy to Lead Demand for Global SMA Market by 2023

Considering the increasing number of infants being diagnosed with spinal muscular atrophy, the spinal muscular atrophy market is expected to expand during the period from 2015 to 2023. On the basis of the symptoms and the age of onset, the SMA market is classified into Type 1 SMA, Type 2 SMA, Type 3 SMA, and Type 4 SMA. Type 1 spinal muscular atrophy is the most severe type and is found in infants aged up to six months. More than 60% of SMA cases are found to be Type 1 SMA, in which infants do not live for more than two years. Type 2 SMA affects children aged from six months to 18 months. Type 3 and 4 SMA are comparatively less severe and carry a prognosis of a normal lifespan. Physiotherapy is the best treatment suggested for SMA when it is at its early stage. 

ISIS-SMNRx is an antisense medicine introduced by Isis Pharmaceutical and has shown positive results in clinical trials. Some other potential candidates are Olesoxime (TRO19622), RO6885247, scAAV9.CB.SMN, RG3039, LMI070, and CK-2127107. Phase 3 study of ISIS-SMNRx for treating Type 1 and Type 2 SMA is expected to be completed by 2016. 

SMA is commonly diagnosed by recognizing mutation in the survival of motor neuron (SMN) gene. Muscle biopsy, nerve condition velocity, and electromyogram are some other ways by which SMA can be diagnosed. AveXis, Inc., F. Hoffmann-La Roche Ltd., Isis Pharmaceuticals, Inc., Cytokinetics, Inc., and Novartis AG are some of the prominent players in the spinal muscular atrophy market.  


At present, there is no well-defined cure for treating spinal muscular atrophy. ISIS-SMNRx is the only drug that is expected to be made available on large scale by 2017. The drug is expected to reach record sales of US$468.2 mn in the U.S. by 2023. 

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