Growing Trends in Spinal Muscular Atrophy Market 2023

According to a new research report released by Transparency Market Research, barring a few drugs, the pipeline for spinal muscular atrophy (SMA) drugs is running dry currently. The few drugs that are present, however, are highly advanced medical technologies progressing through different phases of clinical trials. The key drugs in the spinal muscular atrophy pipeline are RG3039, scAAV9.CB.SMN, CK-2127107, RO6885247, LMI070, Olesoxime (TRO19622), and ISIS-SMNRx.

Ionis Pharma Solution Could Vastly Improve SMA Treatment

ISIS-SMNRx is a drug being developed by Ionis Pharmaceuticals for the treatment of spinal muscular atrophy. As of 2015, it was the only drug that had reached phase 3 of clinical trials. The studies are expected to be completed by July 2017.

“Should this drug be approved for the treatment of spinal muscular atrophy, it will create a highly lucrative opportunity for both Ionis Pharma and Biogen Idec,” states a TMR analyst. “This drug is expected to be released in 2018 and hold high expectations from the healthcare industry for the treatment of spinal muscular atrophy.”

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Growing SMA Carrier Frequency Speeds up Solutions R&D

One of the key drivers affecting the growth of the market for spinal muscular atrophy drugs is the increasing number of carriers of the defective SMA gene. As per the findings revealed by CureSMA, nearly one out of 50 people are carrying the SMA defect. Additionally, this statistic does not include a majority of the global population that for various reasons could not be included in the study. The total percentage of SMA gene carriers could therefore be effectively higher than previously mentioned.

This is creating a greater urge for healthcare and pharmaceutical organizations to create feasible solutions to treat spinal muscular atrophy, and is also pushing the organizations to increase awareness rates of SMA among people.

Lack of Advanced Research Slows Overall Development of SMA Treatments

There are currently multiple research institutes and government agencies that are working together to keep the total number of SMA cases in check. They do not, however, distinguish between the various types of spinal muscular atrophy afflicting the patients. This reduces the accuracy of patient diagnosis and eventually reduces the overall rate of development of treatments for spinal muscular atrophy. At the same time, it makes disease mapping for trends extremely difficult.

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The resultant lack of research is therefore hindering the chances of innovative treatment solutions and the identification of disease progression.

Large Areas Still Untapped for SMA Treatment to Provide Key Opportunities

The U.S. and Europe have designated SMA as an orphan disease. Between the U.S., Europe and Japan, between 25,000 and 50,000 people were diagnosed with spinal muscular atrophy as of June 2015, according to European Pharmaceutical Reviews. The total number of spinal muscular atrophy patients across the world is as yet unknown.

The absence of an approved drug-based treatment for spinal muscular atrophy combined with the unknown number of SMA patients leaves a major unrealized market for SMA drugs that can be tapped into for greater opportunities.

The information presented in this review is based on a Transparency Market Research report, titled, “Spinal Muscular Atrophy Market - Pipeline Assessment, Size, Growth, Trends, and Forecast 2015 - 2023.”

Global Demand for Spinal Muscular Atrophy Market 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.

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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.

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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.