Spinal Muscular Atrophy Market Global Pipeline Overview 2015 to 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.

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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.

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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.

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