SPINAL MUSCULAR ATROPHY MARKET ANALYSIS

Spinal muscular atrophy is a genetic disorder characterized by weakening of the muscles that are involved in movement. It is caused by the loss of motor neurons, which are specialized nerve cells that control muscular movement. The weakening is more severe in muscles close to the center of the body (proximal) than in muscles distant from the center of the body (distal).

Global spinal muscular atrophy market is estimated to be valued at USD 2,013.5 Mn in 2024 and is expected to exhibit a CAGR of 13.3% during the forecast period (2024-2031). 

Figure 1. Global Spinal Muscular Atrophy Market Value (US$ Mn), by Region, 2024

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Increasing prevalence of spinal muscular atrophy is expected to drive growth of spinal muscular atrophy market

The increasing prevalence of spinal muscular atrophy (SMA) is expected to drive the market growth over the forecast period. For instance, according to the Spinal Muscular Atrophy Foundation, a voluntary organization whose mission is to accelerate the development of a treatment for spinal muscular atrophy, SMA is believed to affect around 10,000 to 25,000 children and adults in the U.S., and therefore it is one of the most common rare diseases.

Figure 2. Global Spinal Muscular Atrophy Market Share, By Type, 2024

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Recent Developments

In June 2023, Biogen Inc., a multinational biotechnology company, announced new SPINRAZA (nusinersen) data that will address crucial issues for the spinal muscular atrophy (SMA) community. The data were presented at the SMA Research & Clinical Care Meeting hosted by Cure SMA in Orlando, U.S. SPINRAZA is licensed in over 60 countries to treat newborns, children, and adults with spinal muscular atrophy (SMA). More than 14,000 people throughout the world have received SPINRAZA as a cornerstone of care for SMA.

In March 2023, Novartis, a multinational pharmaceutical corporation, presented new data demonstrating the transformational and long-term benefits of Zolgensma (onasemnogene abeparvovec), a one-time gene therapy for spinal muscular atrophy (SMA). The most recent findings from two Long-Term Follow-Up (LTFU) trials, LT-001 and LT-002, revealed that Zolgensma continues to be efficacious and durable across a wide range of patient demographics, with an overall benefit-risk profile that remains favorable.

In February 2023, Biohaven Ltd., a pharmaceutical company, announced that it had received Fast Track designation from the U.S. Food and Drug Administration (FDA) for taldefgrobep alfa, a new anti-myostatin adnectin for the treatment of spinal muscular atrophy (SMA). Fast Track designation enables critical new pharmaceuticals to reach patients sooner by allowing for more regular discussions with the FDA and expedited review of a drug that treats a serious ailment and meets an unmet medical need.

In August 2020, PTC Therapeutics, Inc., a pharmaceutical company, announced that the U.S. FDA had approved Evrysdi (risdiplam), the first at-home, orally administered treatment for spinal muscular atrophy (SMA) in adults and children aged 2 months and older. Evrysdi demonstrated clinically relevant improvements in motor function and achievement of developmental milestones in two trials involving patients aged two months and up and across all degrees of illness severity, including types 1, 2, and 3.

Increasing product launches are expected to drive the market growth during the forecast period.

Increasing product launches are expected to drive the market growth over the forecast period. For instance, in June 2021, F. Hoffmann-La Roche Ltd, a multinational healthcare company, launched prescription medicine Evrysdi, indicated for the treatment of spinal muscular atrophy (SMA) in adults and children aged 2 months and older, in India.

Global Spinal Muscular Atrophy Market – Impact of Coronavirus (COVID-19) Pandemic

Since the COVID-19 virus outbreak in December 2019, the disease has spread to over 100 countries across the globe and the World Health Organization had declared it a public health emergency on January 30, 2020.

COVID-19 can affect the economy in three main ways: by directly affecting production and demand, by creating disruptions in distribution channels, and through its financial impact on firms and financial markets. Due to nationwide lockdowns, several countries, such as China, India, Saudi Arabia, U.A.E., Egypt, and others are facing problems with regards to the transportation of products from one place to another.

The COVID-19 pandemic had a negative impact on patients with spinal muscular atrophy (SMA) accessing the treatment. For instance, according to an article published by NeurologyLive, delivering quality and relevant information to healthcare professionals treating neurological diseases, in August 2020, in an effort to analyze the impact of COVID-19 on the SMA community, Cure SMA, a voluntary-driven, non-profit organization dedicated solely to eradicating SMA, launched a survey. According to the results of the survey, as of June 1, 2020, 1,513 people had completed the COVID-19 survey, with the majority (65.2%) of these people being parents of an affected person and 32% self-reporting as adults with SMA. Of them, 37.8 percent of them had a healthcare provider (or clinic) cancel or delay an appointment for SMA-related care, and 42.2% had SMA-related services (e.g., physical therapy) delayed or cancelled due to the pandemic. Furthermore, almost one in every five people has had their healthcare practitioner (or clinic) cancel or delay an appointment for SMA treatment, owing to COVID-19

Global Spinal Muscular Atrophy Market: Restraint

The major factor that hinder growth of the global spinal muscular atrophy market include high cost of the treatment. For instance, according to an article published by MJH Life Sciences, largest privately held medical media company in the U.S., in March 2021, the mean per-patient annual direct medical cost was estimated to be between US$ 3,320 (spinal muscular atrophy type 3) in Italy and US$ 324,210 (type 1) in the U.S., with the variability high for other measures as well.

Key Players

Major players operating in the global spinal muscular atrophy market include Biogen, CYTOKINETICS,  F. Hoffmann-La Roche Ltd,  Genentech, Inc.,  PTC Therapeutics, Inc.,  Novartis AG,  Ionis Pharmaceuticals,  Chugai Pharmaceutical Co., Ltd.,  NMD PHARMA A/S,  and Astellas Pharma Inc.