The 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% over the forecast period 2024 - 2031. This high growth can be attributed to growing awareness about spinal muscular atrophy (SMA) conditions and advancement in gene therapy. Furthermore, the launch of novel drugs to treat SMA is also contributing to the market growth.
Market Dynamics:
The rising incidence of spinal muscular atrophy (SMA) conditions and increasing adoption of gene therapy drugs are the major drivers expected to positively influence the growth of the spinal muscular atrophy market over the forecast period. SMA is a relatively common genetic disease, with an incidence rate ranging from 1 in 6,000 to 1 in 10,000 live births. The increasing prevalence of SMA is primarily driven by growing birth rates worldwide. Additionally, gene therapy drugs such as Zolgensma have demonstrated significant clinical benefits in improving SMA conditions. The approval and launch of these drugs has boosted the adoption rate amongst healthcare professionals and patients. However, high treatment cost associated with gene therapy drugs may hamper the market growth to some extent over the forecast period.
Increasing Prevalence of Spinal Muscular Atrophy Globally is driving the Market Growth
SMA is a genetic disorder that affects the nervous system and causes muscle weakening and atrophy. It is one of the leading genetic causes of death for infants. The prevalence of SMA is approximately 1 in 11,000 live births globally. Some countries and regions have a higher incidence rate due to geographical and ethnical factors. For instance, the prevalence of SMA in Asia is reported to be approximately 1 in 6,000 to 10,000 live births. The increasing cases of SMA worldwide is a major factor fueling the growth of the SMA market. As the number of patients diagnosed with the condition rises, the demand for treatment therapies and supportive care products will also increase significantly.
Approval and Launch of Novel Gene Therapies is Proving Highly Beneficial for Market Growth
The approval and launch of novel gene therapies for the treatment of SMA is proving extremely beneficial for market growth. In May 2019, the U.S FDA approved Zolgensma, an AAV9-based gene therapy, for the treatment of SMA in patients less than 2 years of age. It is considered a breakthrough therapy and is one of the most expensive drugs in the world. Several pipeline therapies are also under development such as risdiplam, branaplam, and relusoma that are anticipated to get regulatory approvals in the coming years. These new treatment options offer long-term efficacy, reduced treatment burden and improved quality of life for patients. As a result, their commercial success is fueling significant revenue generation in the spinal muscular atrophy market.
High Treatment Costs Pose a Major Challenge for Market Growth
The high cost of SMA treatment therapies presents a major challenge for market growth. Gene therapies such as Zolgensma have a one-time price tag of US$ 2.1 million, making it one of the most expensive medications available globally. Even chronic supportive care and symptom management is quite costly. Patients require long-term care, physical therapy, nutritional supplementation, ventilator support etc. Moreover, low diagnosis rates in developing nations add to the healthcare burden and out-of-pocket expenses. Due to the high costs involved, SMA treatment remains unaffordable for many patients worldwide. This cost burden is a major restraint on increased product adoption and revenue generation for market players.
Inadequate Diagnosis and Awareness Levels Hamper Early Detection and Treatment
Low diagnosis rates and lack of awareness about SMA impede early detection and treatment initiation, thereby impacting overall market growth. There is a lack of newborn screening programs for SMA in many countries. Patients often remain undiagnosed until symptoms begin to manifest. Diagnosis is also challenging since initial symptoms can mimic other medical conditions. Lack of clinical awareness especially in developing regions results in misdiagnosis. This leads to delayed treatment initiation when the disease has already progressed. Early diagnosis and treatment intervention is crucial for managing SMA. However, inadequate diagnosis and awareness pose a significant market barrier in this regard.
Rising Healthcare Expenditures in Developing Nations Present Lucrative Opportunities
Developing countries present lucrative opportunities for players in the SMA market. Factors such as rising healthcare expenditures, growing per capita incomes, expanding medical insurance coverage and increasing focus on rare disease treatment are expected to drive higher product adoption. Governments are allocating bigger budgets for rare disease diagnosis and care. This will enable more patients to access costly therapies which were previously unaffordable. Pharmaceutical companies can also explore partnerships with local players and invest in new product launches with competitive pricing to leverage opportunities in emerging markets. Overall, developing nations will witness significantly high growth rates in the coming years.
Growing Focus on Novel Therapies such as Gene Editing Techniques Holds Promise
Gene editing techniques such as CRISPR-Cas9 have gathered immense interest from the scientific community for developing SMA therapies. Researchers are exploring ways to leverage these novel approaches for more effective and affordable treatment options. If successful, gene editing can offer curative therapies by directly correcting the faulty SMN1 gene. This will eliminate the need for life-long medications and reduce significant cost burden. Several biotechs are actively conducting preclinical research involving gene editing. The promising potential of these emerging techniques could significantly reshape the SMA treatment landscape and market dynamics in the long run.
Link- https://www.coherentmarketinsights.com/market-insight/spinal-muscular-atrophy-market-1920
Key Development
- In June 2023, Biogen Inc., a global leader in biotechnology, revealed significant new findings regarding SPINRAZA (nusinersen), a key treatment for spinal muscular atrophy (SMA) patients. These findings were unveiled at the SMA Research & Clinical Care Meeting organized by Cure SMA in Orlando, U.S. SPINRAZA holds licenses in over 60 countries worldwide, serving as a crucial therapy for infants, children, and adults grappling with SMA. To date, SPINRAZA has been administered to over 14,000 individuals worldwide, playing an integral role in the standard of care for SMA.
- In March 2023, Novartis, a leading multinational pharmaceutical corporation, unveiled new insights highlighting the enduring and transformative advantages of Zolgensma (onasemnogene abeparvovec), a groundbreaking gene therapy designed for spinal muscular atrophy (SMA). The latest data stemming from two Long-Term Follow-Up (LTFU) trials, LT-001 and LT-002, underscore the sustained effectiveness and resilience of Zolgensma across diverse patient populations. These findings underscore the continued favorable balance between benefits and risks associated with Zolgensma.
- In February 2023, Biohaven Ltd., a pharmaceutical firm, disclosed that its new anti-myostatin adnectin, taldefgrobep alfa, has received Fast Track designation from the U.S. Food and Drug Administration (FDA) for treating spinal muscular atrophy (SMA). Fast Track designation facilitates an accelerated pathway for crucial pharmaceuticals to reach patients promptly. This designation allows for more frequent interactions with the FDA and expedites the review process for drugs addressing serious conditions with unmet medical needs.
- In August 2020, PTC Therapeutics, Inc., a pharmaceutical company, revealed that the U.S. FDA has granted approval for Evrysdi (risdiplam), marking it as the initial at-home treatment for spinal muscular atrophy (SMA) that can be taken orally by adults and children aged 2 months and above. Evrysdi exhibited significant enhancements in motor function and the attainment of developmental milestones in clinical trials involving patients aged two months and older, encompassing all levels of illness severity, including types 1, 2, and 3.
Key Players: Biogen, CYTOKINETICS, F. Hoffmann-La Roche Ltd, Genentech, Inc., PTC Therapeutics, Inc., Novartis AG, Ionic Pharmaceuticals, Chugai Pharmaceutical Co., Ltd., NMD PHARMA A/S, and Astellas Pharma Inc.
