Spinal Muscular Atrophy Treatment Market Segmentation By Treatment (Gene Therapy and Drug); By Route of Administration (Intrathecal and Oral); By Disease Type (Type 1, Type 2, Type 3, and Type 4) - Global Demand Analysis & Opportunity Outlook 2028

  • Report ID: 2724
  • Published Date: Feb 15, 2023
  • Report Format: PDF, PPT

Spinal Muscular Atrophy Treatment  Market Overview

Spinal muscular atrophy (SMA) is a disease that deprives individuals of physical strength by influencing the motor nerve cells in the spinal cord, taking away a person’s ability to walk, eat, or breathe. It is one of the most popular genetic causes of death for infants. This disease is instigated by a mutation in the survival motor neuron gene 1 (SMN1). In a healthy individual, this gene creates a protein that is vital for the function of the nerves that control our muscles. Without this protein, those nerve cells cannot function properly and will eventually die, which can lead to debilitating and sometimes fatal muscle weakness.

According to Cure SMA, this disease affects almost 1 in 11,000 births and nearly 1 in every 50 Americans is a genetic carrier.

There are four major types of spinal muscular atrophy —1, 2, 3, and 4, centered on the age that symptoms commence, and the highest physical milestone attained. People with SMA have trouble performing the basic functions of life, such as breathing and swallowing. Though, spinal muscular atrophy does not affect one’s ability to think, learn, and build relationships with others.

Market Size and Forecast

The spinal muscular atrophy treatment market is expected to observe an attainable high CAGR during the forecast period, i.e., 2020-2028. The market is segmented by treatment, route of administration, disease type, and region. Among the route of administration, the intrathecal segment is expected to hold the dominant position in the market as oral drugs are not ready for commercialization yet. By treatment, the gene therapy segment is still undergoing research which contributes to the dominance of the drug segment in the global market. Spinraza is the only drug approved by the U.S. FDA in December of 2016, for the treatment of spinal muscular atrophy. It was developed by Biogen in partnership with Ionis Pharmaceuticals. The only SMA gene therapy in human trials is Zolgensma, previously known as AVXS-101, which was initially developed by AveXis, now part of Novartis, which is further developing and marketing this gene therapy treatment.

Based on the disease type, type 1 SMA arose as the largest segment of the spinal muscular atrophy treatment market as it’s one of the most common forms. Increasing occurrences of this type and concentration of companies on developing drugs for SMA treatment are the key factors contributing towards the segment’s largest revenue share.CLICK TO DOWNLOAD SAMPLE REPORT

Growth Drivers

Extensive research going on to produce new ways of treating the disease

Zolgensma was approved by the U.S. Food and Drug Administration in 2019 to attend all types of SMA in newborns and children up to age 2 over a single intravenous route of administration. This approval marks Zolgensma, as the second disease-modifying treatment for spinal muscular atrophy, and its first gene therapy treatment.

Risdipalm (formerly RG 7916) and RG 6083 (Olesoxime) are among the few potential therapies, in which Olesoxime has shown very disappointing results in a long term Phase-2 trial by Roche.

There are several other products in the clinical pipeline from companies such as Cytokinetics, Novartis, Voyager Therapeutics, and BioBlast Pharma.

Various organizations working tirelessly to raise awareness about rare diseases

CureSMA is a voluntary-driven organization committed to eradicating SMA by funding and supporting comprehensive research to lead the way for a cure and enhance the treatment and care for SMA patients and their families. Further, the SMA Foundation and Muscular Dystrophy Association, are some other active organizations creating awareness about this disorder.

Furthermore, a rise in disposable income, a surge in healthcare facilities in developing nations, and awareness for rare diseases are estimated to drive the market in the forecast period.


High Cost of Products to Impede Market Expansion

The cost-intensive products coupled with high prices accompanying the rare disease therapies will consequently hinder the growth of the market. It will restrict the adoption of treatment for many patients, which consecutively will act as a major constraint for the spinal muscular atrophy treatment market revenue.

Market Segmentation

Our in-depth analysis of the spinal muscular atrophy treatment  market includes the following segments:

By Treatment

  • Gene Therapy
  • Drug

By Route Of Administration

  • Intrathecal
  • Oral

 By Disease Type

  • Type 1
  • Type 2
  • Type 3
  • Type 4

By Region

Geographically, the spinal muscular atrophy treatment market is segmented into five major regions including North America, Europe, Asia Pacific, Latin America, and the Middle East & Africa region.

North America is projected to hold the largest market share during the forecast period. The dominance is owed to obvious factors such as the first drug being manufactured and approved in the region, in 2016 and continuous R&D concerning new drug and gene therapy being conducted in the region. Further, high healthcare expenditure, amplified cases of SMA patients in this region, high awareness among the populace regarding the disease, and several active organizations and campaigns working tirelessly to raise awareness and contributes to the society, are some other key factors contributing towards the regional growth.

The European region is anticipated to hold the second-largest market share in the spinal muscular atrophy treatment market. The growth in this region is attributed to the rising incidences of spinal muscular atrophy patients and rising government spending for the healthcare facilities in the region.

Asia-Pacific is estimated to grow at the fastest rate during the forecast period. Factors such as a rise in the incidence of spinal muscular atrophy disorders, upsurge in healthcare expenditure, penetration of leading market players, growing populace, and a rise in disposable income, are projected to propel the growth in the Asia-Pacific region.

The spinal muscular atrophy treatment  market is further classified based on region as follows:

  • North America (U.S. & Canada) Market size, Y-O-Y growth & Opportunity Analysis
  • Latin America (Brazil, Mexico, Argentina, Rest of Latin America) Market size, Y-O-Y growth & Opportunity Analysis
  • Europe (U.K., Germany, France, Italy, Spain, Hungary, Belgium, Netherlands & Luxembourg, NORDIC, Poland, Turkey, Russia, Rest of Europe) Market size, Y-O-Y growth & Opportunity Analysis
  • Asia-Pacific (China, India, Japan, South Korea, Indonesia, Malaysia, Australia, New Zealand, Rest of Asia-Pacific) Market size, Y-O-Y growth & Opportunity Analysis
  • the Middle East and Africa (Israel, GCC (Saudi Arabia, UAE, Bahrain, Kuwait, Qatar, Oman), North Africa, South Africa, Rest of the Middle East and Africa) Market size, Y-O-Y growth & Opportunity Analysis.

Top Featured Companies Dominating The Market


In the News

April 28, 2020: Roche’s developed Risdiplam drug has shown noteworthy improvement in survival and motor milestones in infants testing who was diagnosed with Type 1 spinal muscular atrophy (SMA).

Roche is leading the clinical development of Risdiplam, an investigational and orally administered survival motor neuron-2 (SMN2) splicing modifier for Spinal Muscular Atrophy patients. This is developed as part of a collaboration with the SMA Foundation and PTC Therapeutics.

March 2020: Reldesemtiv, by Cytokinetics, Incorporated, Won  European Medicines Agency (EMA) orphan drug designation for the treatment of ALS. Previously this drug was approved as an orphan drug designation for the treatment of ALS by the U.S. Food & Drug Administration. Reldesemtiv (formerly CK-2127107) is an investigational therapy that might recover muscle function and physical performance in patients diagnosed with spinal muscular atrophy (SMA). It is being developed in collaboration with Astellas.

Author Credits:  Radhika Gupta, Shivam Bhutani

  • Report ID: 2724
  • Published Date: Feb 15, 2023
  • Report Format: PDF, PPT

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