According to Nova one advisor, the global Spinal Muscular Atrophy Treatment market was valued at USD 3.4 Billion in 2021 and it is expected to hit around USD 18.9 billion by 2030 with a CAGR of 18.7% during the forecast period 2022 to 2030.
According to Nova one advisor, the global Spinal Muscular Atrophy Treatment market was valued at USD 3.4 Billion in 2021 and it is expected to hit around USD 18.9 billion by 2030 with a CAGR of 18.7% during the forecast period 2022 to 2030.
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Key Takeaways:
- By type, the type-1 segment held the largest market share in 2021 due to higher prevalence and wide availability of products for the treatment of patients with type-1 spinal muscular atrophy
- By treatment, the gene therapy segment is expected to be the fastest growing segment over the forecast period due to increase market penetration. Zolgensma is the only approved gene therapy in 37 countries to treat patient with SMA
- By drug, the Spinraza segment dominated the market in 2021 due to presence of supportive reimbursement policies and increasing awareness about products. Currently, Spinraza is applicable for full reimbursement in three European countries Italy, Norway, Netherlands
- By route of administration, the injection segment dominated the market in 2021 due to high safety, efficacy, and tolerability associated with infusion drugs to SMA patients through this route of administration
- Asia Pacific is expected to be the fastest growing region during the forecast period. The growth of region is attributable to the entry of new products into the region. For instance, in July 2021, F. Hoffmann-La Roche Ltd launched Evrysdi (risdiplam) for the treatment of patient with spinal muscular atrophy in India
This growth can be attributed to the increasing product approval and launches. For instance, in August 2020, the Food and Drug Administration approved Evrysdi (risdiplam) developed by Genentech, Inc., for the treatment of pediatric patients aged two months and older with spinal muscular atrophy (SMA).
In addition, in May 2020, Novartis AG received conditional approval to its SMA candidate Zolgensma (onasemnogene abeparvovec) from the European Commission (EC) to treat patients with SMA. It is only approved gene therapy in Europe. In March 2021, PTC Therapeutics, Inc., received marketing approval for Evrysdi (risdiplam) from the EMA for the treatment of patient with type1, 2, and 3 spinal muscular atrophy. Thus, increasing number of product approvals is expected to drive the spinal muscular atrophy treatment market.
Novartis AG is supporting SMA patients by offering medicine at free of cost through global Managed Access Program (MAP). This program provides 100 doses of Zolgensma at free of charge to eligible SMA patients across continents including North America, South America, Asia, Australia, Europe, and Africa. Thus, presence of such supportive programs for Zolgensma strengthens promotional activities and also aid in generating revenue.
Global SMA newborn screening and easy access to diagnostic tools should be included in routine approach. This would ensure early diagnosis and timely access to treatment. For instance, in July 2021, a charlotte baby received a breakthrough gene therapy treatment for Spinal Muscular Atrophy (SMA) at UNC Medical Center in Chapel Hill, N.C., after diagnosis with spinal muscular atrophy through Early Check newborn screening program. Such screening programs are expected to boost early diagnosis and treatment of disease.
Moreover, there are several extensive research collaborations undertaken by pharmaceutical companies with research institutes for developing novel therapies for the treatment of spinal muscular atrophy patients. For instance, in March 2021, PTC Therapeutics, Inc., and The Spinal Muscular Atrophy Foundation entered into research collaboration to advance scientific research and developing new treatment to treat patients with SMA.
However, high costs associated with the treatment and low availability of participant for clinical trial purposes may restrain market growth over the forecast period. The high cost related to research and development of an orphan drug is a major factor of rising prices of product. For instance, the most commonly used drug Spinraza costs around USD 125,000 per injection and USD 750,000 annually. Furthermore, Zolgensma (AVXS-101) costs USD 2.1 million for one time treatment.
Report Scope of the Spinal Muscular Atrophy Treatment Market
| Report Coverage | Details |
| Market Size | USD 18.9 Billion by 2030 |
| Growth Rate | CAGR of 18.7% from 2022 to 2030 |
| Largest Market | North America |
| Fastest Growing Market | Asia Pacific |
| Base Year | 2021 |
| Forecast Period | 2022 to 2030 |
| Segments Covered | Type, treatment, drug, route of administration, and Region, |
| Companies Mentioned | 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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Type Insights
The type1 segment dominated the market and accounted for the largest revenue share of 63.8% 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.
Treatment Insights
The drug segment dominated the market and accounted for the largest revenue share of 75.11% 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.
Drug Insights
The Spinraza segment dominated the market and accounted for the largest revenue share of 50.9% 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.
Route Of Administration Insights
The injectable segment dominated the market and accounted for the largest revenue share of 73.10% 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.
Regional Insights
North America dominated the market and accounted for revenue share of 73.9% 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.
Some of the prominent players 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
- NMD PHARMA A/S
Segments Covered in the Report
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-segments from 2017 to 2030. For this study, Nova one advisor, Inc. has segmented the global Spinal Muscular Atrophy Treatment market
- Type
- Type 1
- Type 2
- Type 3
- Type 4
- Treatment
- Gene Therapy
- Drug
- Drug
- Spinraza
- Zolgensma (AVXS-101)
- Evrysdi
- Others
- Route of Administration
- Oral
- Injection
By Region
- North America
- Europe
- Asia Pacific
- Latin America
- Middle East & Africa
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