Spinal muscular atrophy (SMA) is a group of hereditary autosomal recessive neuromuscular disorders that causes degeneration of motor neurons resulting in muscular atrophy and weakness. SMA afflicts approximately 1 out of every 11,000 live births and is the leading genetic cause of death for infants. SMA therapeutics work by targeting the underlying genetic defect caused due to mutations in the survival motor neuron 1 (SMN1) gene or by increasing the level of functional SMN protein.
The global SMA market is estimated to be valued at US$5 billion in 2024 and is expected to exhibit a CAGR of 13% over the forecast period 2024.
Key Takeaways
Key players operating in the spinal muscular atrophy market are Biogen Inc., Novartis AG, Genentech, and Roche. Biogen's Spinraza was the first drug approved for treatment of SMA and remains the dominant treatment option. With the approval of Novartis' gene therapy Zolgensma in 2019, a new era in SMA treatment began, offering a one-time potentially curative option. The demand for SMA therapeutics is increasing rapidly due to growing diagnosis of SMA cases and ongoing clinical trials evaluating several pipeline drug candidates. Furthermore, collaborations between pharma companies and regulatory approvals of new drugs are helping expand the global SMA treatment market.
The growing diagnosis of SMA cases, especially in infant populations, is significantly driving the demand for effective treatment options. With newborn screening programs for SMA being implemented across several countries, thousands of pre-symptomatic cases are being identified annually, requiring lifesaving treatment interventions. Spinal Muscular Atrophy Market demand is high-impact clinical trials also demonstrated significant patient benefits, encouraging widespread treatment uptake.
The market is witnessing rapid global expansion driven by the commercialization of novel one-time gene therapy and ongoing regulatory reviews. While North America and Europe currently dominate the market, regions like Asia Pacific and Latin America are expected to offer high future potential owing to rising healthcare investment and access to innovative therapies. Promising drug pipelines and established collaboration models are likely to support market access and growth in these emerging economies over the coming years.
Market key trends
One of the key trends witnessed in the Spinal Muscular Atrophy Market Growth is the availability of first-ever disease modifying, potentially curative treatment options. Gene therapies like Zolgensma are transforming SMA treatment from a chronic symptomatic approach to a single-dose potential cure. Other promising pipeline candidates like Ensysce Biosciences' drug candidate and Vyant Bio's intrathecal gene therapy also employ gene augmentation or gene editing approaches offering hope of a functional cure. As more disease modifying therapies obtain regulatory approvals, SMA is expected to transition from a fatal genetic disorder to a highly manageable condition.
Porter's Analysis
Threat of new entrants: High capital requirements and expensive clinical trails pose barrier for new players.
Bargaining power of buyers: Large pharmaceutical companies can negotiate on price with manufacturers.
Bargaining power of suppliers: Suppliers have limited control due to availability of alternative raw material sources.
Threat of new substitutes: Emerging gene therapy offers potential substitute treatment.
Competitive rivalry: Intense competition exists between established pharmaceutical manufacturers.
Geographical Regions
North America accounts for the largest share in the global spinal muscular atrophy market in terms of value due to high healthcare expenditure and presence of advanced healthcare infrastructure.
Asia Pacific is poised to growth at the fastest rate during the forecast period owing to increasing patient population, rising awareness about the disease, and improving healthcare facilities. Countries such as China and India are expected to contribute significantly to the market growth.
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