Zolgensma Global Market Report 2025: Epidemiology, Pipeline Analysis, Market Insights & Forecasts

The zolgensma market research report is one of a series of new reports that provides zolgensma market statistics, including the zolgensma industry global market size, regional shares, competitors with the zolgensma market share, detailed zolgensma market segments, market trends, and opportunities, and any further data you may need to thrive in the zolgensma industry. This zolgensma market research report delivers a complete perspective of everything you need, with an in-depth analysis of the current and future scenarios of the industry.

The growth in the forecast period can be attributed to rising interest in personalized medicine approaches, increasing global healthcare spending, growth in telemedicine, increasing focus on rare disease treatment, and growth in digital health technologies. Major trends in the forecast period include advancements in gene therapy technologies, technological advancements in manufacturing processes, development of new biomarkers, advancements in digital health technologies, and development of intrathecal formulations.

The rising incidence of spinal muscular atrophy (SMA) is expected to drive the growth of the Zolgensma market in the coming years. SMA is a genetic disorder that causes the degeneration of motor neurons in the spinal cord, leading to progressive muscle weakness, particularly in infants and young children. This condition can result in severe physical disabilities and life-threatening complications. Improved genetic testing and newborn screening programs have contributed to the increased identification of SMA cases, allowing for earlier and more accurate diagnoses. Zolgensma addresses the genetic cause of SMA by delivering a functional copy of the SMN1 gene, which helps restore the production of the survival motor neuron (SMN) protein essential for motor function and muscle strength. This treatment has shown promising results in improving motor skills and survival in affected children. For example, in August 2023, the National Health Service England reported that about 70 children are born with SMA each year in the UK. By November 2022, 118 out of 161 patients with SMA Type 1 were over the age of two and living without permanent ventilatory support, showcasing the potential of Zolgensma in improving patient outcomes.

The increasing prevalence of rare diseases is also expected to contribute to the growth of the Zolgensma market. Rare diseases, which affect a small percentage of the population, often pose challenges in diagnosis and treatment. The rise in rare diseases can be attributed to advances in diagnostic technologies and greater awareness, both of which have led to earlier detection of conditions that were previously underdiagnosed. Zolgensma, which targets rare diseases such as spinal muscular atrophy, helps by providing a functional copy of the defective SMN1 gene, enabling the production of the essential SMN protein that supports motor neuron survival and muscle function. In September 2024, the European Union reported that there are over 6,000 distinct rare diseases affecting up to 36 million people across the EU, further emphasizing the need for treatments such as Zolgensma for rare conditions.

The key factor driving the growth of the Zolgensma market is strategic investments aimed at expanding production capabilities, broadening geographic access, and accelerating research and development for innovative gene therapies. These investments are crucial in supporting increased demand, advancing gene therapy innovations, expanding access to treatments globally, and improving supply chain efficiency. In April 2022, Novartis AG received approval from the U.S. Food and Drug Administration for its Durham, North Carolina facility, which is dedicated to multi-product gene therapy manufacturing. This 170,000-square-foot facility is now able to produce, test, and release commercial Zolgensma, as well as manufacture gene therapy products for ongoing and future clinical trials. This expansion is part of a broader strategy to support the growing need for gene therapies and enhance global access to cutting-edge treatments.

The key company operating in the zolgensma market is Novartis AG

North America was the largest region in the zolgensma market in 2024. The regions covered in zolgensma report are Asia-Pacific, Western Europe, Eastern Europe, North America, South America, Middle East and Africa.

The countries covered in the zolgensma market report are Australia, India, China, South Korea, Japan, UK, France, Germany, USA, Canada.

Zolgensma is a gene therapy developed to treat spinal muscular atrophy (SMA), a rare genetic disorder that causes muscle weakness and loss of motor function. The therapy works by replacing the defective or missing SMN1 gene in children under the age of two, which helps restore motor function and improve survival outcomes.

Note that the outlook for this market is being affected by rapid changes in trade relations and tariffs globally. The report will be updated prior to delivery to reflect the latest status, including revised forecasts and quantified impact analysis. The report’s Recommendations and Conclusions sections will be updated to give strategies for entities dealing with the fast-moving international environment.

The primary indications for Zolgensma include spinal muscular atrophy (SMA) type 1, SMA type 2, SMA type 3, and pre-symptomatic SMA. SMA type 1 is the most severe form of the disease, characterized by the rapid degeneration of motor neurons in the spinal cord, leading to progressive muscle weakness and atrophy. Zolgensma is used in pediatric populations (infants and children) as well as adults and is distributed through various channels, including hospitals, specialty clinics, and pharmacies.

The zolgensma market consists of infusion sets, syringes, and other medical supplies for intravenous administration. Values in this market are ‘factory gate’ values, that is the value of goods sold by the manufacturers or creators of the goods, whether to other entities (including downstream manufacturers, wholesalers, distributors and retailers) or directly to end customers. The value of goods in this market includes related services sold by the creators of the goods.

The market value is defined as the revenues that enterprises gain from the sale of goods and/or services within the specified market and geography through sales, grants, or donations in terms of the currency (in USD, unless otherwise specified).

The revenues for a specified geography are consumption values and are revenues generated by organizations in the specified geography within the market, irrespective of where they are produced. It does not include revenues from resales along the supply chain, either further along the supply chain or as part of other products.

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