What are the major factors driving the growth of the Spinal Muscular Atrophy Treatment market?

In the year 2025, the industry size of spinal muscular atrophy treatment is estimated at USD 6.82 billion. Read More

What would be the CAGR of Spinal Muscular Atrophy Treatment Market over the forecast period?

Spinal Muscular Atrophy Treatment Market size was over USD 5.9 billion in 2024 and is anticipated to cross USD 53.02 billion by 2037, witnessing more than 18.4% CAGR during the forecast period i.e., between 2025-2037.. Read More

Which region will provide more business opportunities for growth of Spinal Muscular Atrophy Treatment market in future?

The major players in the market are PTC Therapeutics, Voyager Therapeutics, Inc, Astellas Pharma Inc., Novartis AG, Pfizer, Inc., F. Hoffmann-La Roche Ltd, CYTOKINETICS, INC., Ionis Pharmaceuticals, Inc., Regeneron Pharmaceuticals, Inc, and others. Read More

Spinal Muscular Atrophy Treatment Market Trends

Growth Drivers

Increased government support for rare disease treatments: More than 7,000 rare diseases affect more than 30 million people in the U.S. Support from the government for rare disease treatments, including SMA, is acting as a significant driver for the spinal muscular atrophy treatment market. Governments in many countries are offering incentives such as grants, tax credits, and funding for R&D to encourage the development of therapies for rare diseases. For instance, in April 2024, the Canadian Institutes of Health Research Rare Disease Research Initiative provided UBC Faculty of Medicine researchers with USD 20 million in federal funding, for creating RareKids-CAN: Pediatric Rare Disease Clinical Trials and Treatment Network.
Rise in rate of diagnosis of SMA in infants and children: This is a crucial spinal muscular atrophy treatment market driver due to the implementation of newborn screening programs and technological advancements. Many countries have begun including SMA in their routine newborn screening, allowing for earlier detection and intervention. The Australian Government Department of Health and Aged Care published an article in October 2023 related to SMA therapies. It states that the Albanese Government offers families extended access to life-changing gene therapy for SMA through the Pharmaceutical Benefits Scheme (PBS).

Challenges

The low success rate in clinical trials: Developing novel therapies for SMA often involves complete biological processes, particularly in gene and RNA-based therapies, where achieving consistent efficacy can be difficult. Many promising treatments fall in late-stage trials due to unexpected safety issues or insufficient therapeutic benefits. This further leads to significant financial losses and delays in bringing new treatments to market. This not only discourages smaller firms but also increases the overall cost and timeline for drug development.
Stringent regulatory approval for new therapies: This presents a considerable barrier in the SMA treatment market. Regulatory bodies such as the FDA and EMA impose rigorous standards to ensure the safety and efficacy of new treatments, particularly for advanced therapies. The complex approval process requires extensive clinical data, long-term studies, and post-marketing surveillance. These regulatory hurdles can also limit the market entry of innovative treatments, especially I countries with less flexible approval frameworks.

Spinal Muscular Atrophy Treatment Market: Key Insights

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.

Restraints

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.

Spinal Muscular Atrophy Treatment Market Trends