Spinal muscular atrophy or SMA is a genetic disorder that affects the nervous system, impairing voluntary muscle movement. It is a hereditary autosomal recessive disease and causes mortality in children. Earlier onset of SMA makes the impact greater, damaging the motor function of the child to a larger extent. Children who show symptoms at birth or infancy have type 1 SMA, which leads to minimal motor skill functioning. However, the ones who develop it as children contract type 2 and 3 global SMA market and the ones who get it in teenage or as adults have type 4 SMA. These latter patients have a slightly better ability to maintain their motor skills than the type 1 cases.
Spinal muscular atrophy can be applied to multiple conditions and its severity can be segmented into four types: Type I being the most severe, Types II and III sequentially less severe, and Type IV that affects adults. It is primarily caused by the rapid degradation of the motor neurons between the spine and the brain, and a patient loses control of his/her muscles over time. Due to the severe nature of this disease and the growing number of patients suffering from spinal muscular atrophy, medical organizations are mounting up their efforts to help cure it.
How Serious of a Threat is SMA Currently?
According to CureSMA, roughly one per fifty people carry the defective genes responsible for spinal muscular atrophy in newborns. This estimate does not, however, carry across the global population. Therefore the total number of SMA gene carriers may be even higher. This is certainly a serious conundrum as there is currently no solid cure for spinal muscular atrophy. The pipeline drugs for spinal muscular atrophy are thus under intensive development to answer for the growing number of patients in the world.
For the severity of this disease, relatively insufficient research and development efforts are being taken on a global front. This applies especially to the exclusion of spinal muscular atrophy in a segregated format during research, thus limiting the scope of research. Currently, the treatments to spinal muscular atrophy focus on the symptoms rather than the problem itself, something that upcoming pipeline drugs are promising to resolve.
What are the More Prominent Drugs in the Pipeline?
Till the beginning of 2016, only one drug was present in phase 3 of the pipeline for spinal muscular atrophy drugs: Ionis-SMNrx, formerly known as ISIS-SMNrx, and owned by Ionis Pharmaceuticals. The drug was enrolled in phase 3 of trials in 2014 for studying its efficacy and safety patients suffering from Type I and Type II spinal muscular atrophy. The clinical trials are expected to be finished in mid-2017 and the drug will be released in 2018. Once released, it is expected to dominate the market for spinal muscular atrophy treatments.
Other drugs in the SMA pipeline include Olesoxime by Trophos SA, LMI070 by Novartis International AG, RO6885247 by Roche, and CK-2127107 by Cytokinetics and Astellas Pharma.
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What are the Levels of Involvement in SMA Pipeline from Key Regions?
At the moment, North America is touted as the leading region in terms of research and development as well as market attractiveness. One of the key factors that contribute to this is the presence of some of the top pharmaceutical companies in the world that are intensifying their research and development efforts on the treatment of spinal muscular atrophy. Additionally, North America holds a higher density of SMA patient population, further increasing the demand for its treatment in the region.
Furthermore, the governing bodies in North America are showing a high level of support for research on SMA. The governments provide substantial incentives for the development of orphan drugs. The regulatory policies of the region are also favorable to the development of new drugs for serious ailments such as SMA. Europe also shares a high level of development in the SMA pipeline, while Asia Pacific and the rest of the world have not yet shown significant strides in it.
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