The global spinal muscular atrophy treatment market size was valued at USD 3,882.0 million in 2021 and is expected to witness a compounded annual growth rate (CAGR) of 18.6% from 2022 to 2030. The market is primarily driven by rising incidence of spinal muscular atrophy (SMA). According to the National Organization for Rare Disorders (NORD), spinal muscular atrophy (SMA) affects almost 1 in 10,000 people globally. Thus, rising prevalence of SMA and the approval for gene therapy for SMA treatment is one of the major factors in generating revenue for the market. A multidisciplinary standard therapy approach is used for the treatment of patients with SMA which includes, symptomatic therapy such as physical, occupational, and respiratory function monitoring and disease-modifying therapies. The U.S. Food and Drug Administration (FDA) approved world’s first disease modifying therapy, Spinraza. It has shown positive promising results with improved survival rates in patients. Thus, approval of new disease-modifying therapies is anticipated to drive market growth.
Moreover, rising awareness regarding novel therapies is a key factor that contributes largely to the market growth. For instance, in May 2021, Cure SMA organized the 2022 Annual SMA Conference in Anaheim to reunite the SMA community including researchers, patients and their caregivers, and clinicians. Under this conference, a wide variety of activities were performed including education workshops, keynote sessions for researchers, and social activities. Such conferences provide opportunities for manufacturers to connect and interact with SMA people and understand their unmet treatment needs.
The development of new products is challenging task for new players due to low success rate in clinical trials. According to the National Library of Medicine, the clinical trial success rate is 10% while developing new SMA drugs. For instance, in December 2020, F. Hoffmann-La Roche Ltd stopped clinical development program for Olesoxim, as the product failed to provide benefits to type 2 & 3 SMA patients. Thus, low success rate in clinical trials create uncertainty among new players to invest in new drug development, thereby, restraining market growth.
The type1 segment dominated the market and accounted for the largest revenue share of 63.1% in 2021 owing to the high prevalence rate. It is one of the most common types of SMA affecting approximately 60.0% of total infants born with SMA. The increasing focus of companies on developing novel drugs for the treatment of patients with SMA is projected to fuel market growth. Several therapies have been approved by FDA for the treatment of type-1 patients including Zolgensma (onasemnogene abeparvovec-xioi) in 2019.
In addition, the type-1 segment is expected to show lucrative growth during the forecast period. This growth is attributed to the presence of a strong product pipeline and increasing research activities. For instance, in March 2022, Novartis AG announced the result of the SPRINT study to assess the effect of pre-symptomatic administration of Zolgensma in patients. The company also published post-hoc data from its STR1VE-EU, START and STR1VE-US studies in SMA type 1 patients, which revealed that the patients could swallow, speak and maintain airway function. This result is anticipated to increase the prescription rate for this drug by encouraging physicians to prescribe this drug to type-1 patients, thereby, driving the segment growth.
The drug segment dominated the market and accounted for the largest revenue share of 75.9% in 2021 owing to the wide availability of products. Currently, two drugs are approved by the FDA for the treatment of SMA including Spinraza (nusinersen) and Evrysdi (risdiplam). Low cost associated with research and development of drugs and ease of accessibility compared to gene therapy treatment is driving the segment growth.
Gene therapy segment is anticipated to grow at the fastest CAGR of 33.1% during the forecast period owing to its increasing penetration for spinal muscular atrophy treatment. Launch of a novel gene therapy product for managing symptoms associated with SMA augments market growth. For instance, Zolgensma developed by Novartis AG is the only FDA approved gene therapy product used for controlling disease progression.
The Spinraza segment dominated the market and accounted for the largest revenue share of 50.0% in 2021. Spinraza is a prescription medicine used to treat pediatric and adult patients. It is administrated through intrathecal injection by experienced healthcare professionals. Spinraza is fully reimbursed worldwide by some countries such as Italy, Norway, Netherlands, Romania, Croatia, and Poland. Thus, the presence of supportive reimbursement policies is propelling the segment growth.
The evrysdi drug segment is expected to witness significant growth over the forecast period. This drug is expected to improve the survival rate in patients with SMA ages 2 months of age and older. It was designated an orphan medicine by the FDA in 2019. Currently, a number of public and private insurance companies offer coverage for Evrysdi for the treatment of patients with SMA including Highmark (Medicare), Aetna, Anthem, BCBS Alabama, Cigna, Florida Blue (BCBS FL), HealthPartners, United Healthcare, Colorado, Connecticut, and HCSC. The availability of insurance coverage encourages practitioners to prescribe this drug patients, thereby, increasing the adoption of Evrysdi.
The injectable segment dominated the market and accounted for the largest revenue share of 73.7% in 2021. This can be attributed to the owing to high target specificity and efficacy associated with this route of administration. The majority of the approved SMA drugs are administrated intravenously by experienced practitioners. For instance, SPINRAZA is administered intrathecally to patients with recommended dose of 12 mg (5mL) per suspension. Furthermore, Zolgensma is also administered intravenously to the patient dose of 5.5ml.
Oral segment is expected to be the fastest growing segment over the forecast period owing to advantages associated with it such as non-invasiveness, ease of digestion, pain avoidance, versatility, and low side-effects compared to other route of administration methods. Currently, Evrysdi is only the FDA approved orally administrated drug for the treatment of pediatric patients with SMA.
North America dominated the market and accounted for revenue share of 73.6% in 2021. Mounting prevalence of SMA disorders and increased spending on treatment products are the main factors for the dominance of the region. For instance, around 10,000 to 25,000 children and adults are living with SMA in U.S. In addition, according to SMA Canada, approximately 700 people are living with this condition in Canada as of July 2022 and 30 patients are currently receiving Spinraza.
In Asia Pacific, the market is expected to witness a CAGR of 35.9% during the forecast period. The growth of the region is attributable to the entry of new players in the region. For instance, in March 2020, the Japanese Ministry of Health, Labour and Welfare (MHLW) approved a gene therapy candidate Zolgensma (onasemnogene abeparvovec) developed by Novartis AG for the treatment of the pediatric patients in Japan. This approval is expected to have a positive impact on regions market growth.
The leading players are concentrating on growth strategies, such as licensing agreements to gain market share. For instance, in January 2022, Biogen and Ionis Pharmaceuticals, Inc., enter into licensing agreement for the development of another experimental drug BIIB115 (an antisense oligonucleotide) for the treatment of patients with SMA. BIIB115 is an experimental preclinical-stage drug. This agreement strengthens Biogen’s SMA drug portfolio. Some of the prominent players in the global spinal muscular atrophy treatment market include:
Ionis Pharmaceuticals Inc.
F. Hoffmann-La Roche Ltd
Scholar Rock, Inc.
NMD PHARMA A/S
Market size value in 2022
USD 4,601.8 million
Revenue forecast in 2030
USD 18.0 billion
CAGR of 18.6% from 2022 to 2030
Base year for estimation
2018 - 2020
2022 - 2030
Revenue in USD million and CAGR from 2022 to 2030
Revenue forecast, company ranking, competitive landscape, growth factors, and trends
Type, treatment, drug, route of administration, region
North America; Europe; Asia Pacific; Latin America; MEA
U.S.; Canada; U.K.; Germany; France; Spain; Italy; China; Japan; India; Australia; South Korea; Brazil; Mexico; Argentina; South Africa; Saudi Arabia; UAE
Key companies profiled
Biogen; Novartis AG; Ionis Pharmaceuticals Inc.; Biohaven Pharmaceuticals; F. Hoffmann-La Roche Ltd; Cytokinetics; Scholar Rock, Inc.; PTC Therapeutics; NMD Pharma A/S
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This report forecasts revenue growth at global, regional, and country levels and provides an analysis of the latest industry trends in each of the sub-markets from 2018 to 2030. For this study Grand View Research has segmented the global spinal muscular atrophy treatment market report on the basis of type, treatment, drug, route of administration, and region:
Type Outlook (Revenue, USD Million, 2018 - 2030)
Treatment Outlook (Revenue, USD Million, 2018 - 2030)
Drug Outlook (Revenue, USD Million, 2018 - 2030)
Route of Administration Outlook (Revenue, USD Million, 2018 - 2030)
Regional Outlook (Revenue, USD Million, 2018 - 2030)
b. The global spinal muscular atrophy treatment market size was estimated at USD 3,882.0 million in 2021 and is expected to reach USD 4,601.8 million in 2022.
b. The global spinal muscular atrophy treatment market is expected to witness a compound annual growth rate of 18.6% from 2022 to 2030 to reach USD 18.0 billion in 2030.
b. Based on type, type 1 segment held the largest share of 61.72% in 2021, owing to the increasing incidence and prevalence, and availability of medicines for the treatment of type 1 SMA.
b. Some key players operating in the spinal muscular atrophy treatment market include Biogen; Novartis AG; Ionis Pharmaceuticals Inc.; Biohaven Pharmaceuticals, F. Hoffmann-La Roche Ltd; Cytokinetics; Scholar Rock, Inc.; PTC Therapeutics; and NMD Pharma A/S.
b. Key factors driving the spinal muscular atrophy treatment market growth include the increasing prevalence of diseases, increasing product approval and launches, rising initiatives for creating awareness of SMA, and supportive regulatory and reimbursement policies.
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