The global spinal muscular atrophy treatment market size is expected to expand at a significant CAGR during the forecast period, 2021–2028. The growth of the market is attributed to the increasing number of initiatives for the treatment of rare diseases and growing awareness about spinal muscular atrophy.
Spinal muscular atrophy (SMA) is a group of inherited diseases which affects the functioning of muscles due to deterioration. It results into weakness and may lead to death. Genetic disorders such as mutation or deletion causes SMA. It affects motor neurons which are present in the spinal cord and brain. SMA is categorized into type1, type 2, type 3, and type 4 based on symptoms and causes which are observed. The SMA treatment includes drugs and gene replacement surgery or therapy.
There are several public and non-profit organizations who are creating awareness about SMA which helps the market to grow. The National Institute for Health and Care Excellence (NICE), in January 2018, invited Biogen for submitting a single technology appraisal of Spinraza to allow National Health Services (NHS) funding. Both NHS and NICE are evolving managed access agreement for Biogen’s Spinraza for long-term reimbursement plan.
Moreover, Muscular Dystrophy Association, SMA Foundation, and Cure SMA are generating awareness about spinal muscular atrophy. Key companies are largely investing into research and development of treatment. Also, research institutes are concentrating on the treatment development for this disorder. The US Food and Drug Administration (FDA) approved an orphan drug license to RG7916. This was a combined development program by Roche, SMA Foundation, and PTC Therapeutics for the SMA treatment.
Market Trends, Drivers, Restraints, and Opportunities
- Growing prevalence of spinal muscular atrophy is major factor responsible for the market growth over the forecast period.
- Increasing awareness about treatment and diagnosis of SMA, rising number of government initiatives, and large pool of patients are projected to fuel the market expansion.
- Technological advancement and rising research and development activities are anticipated to boost the expansion of the market in the coming years.
- Increasing approvals of the U.S. FDA for the development of novel drugs and rising initiatives for improvement in healthcare infrastructure are estimated to drive the growth of the market.
- Emerging treatment therapies and improvement in reimbursement scenario are expected to propel the market growth in coming years.
- High cost of treatment for SMA is major factor responsible to hamper the market growth over the forecast period.
- Lack of experienced professionals and lower adoption rate of SMA treatment are expected to impede the market expansion in coming years.
Scope of Spinal Muscular Atrophy Treatment Market
The report on the global spinal muscular atrophy treatment market includes an assessment of the market, trends, segments, and regional markets. Overview and dynamics have also been included in the report.
Spinal Muscular Atrophy Treatment Market - Global Industry Analysis, Growth, Share, Size, Trends, and Forecast
Routes of Administration (Oral and Intrathecal), Treatments (Gene Therapy, Drug [Spinraza, RG6083, and RG7916]), Disease Types (Type I, Type II, Type III, and Type IV)
Asia Pacific, North America, Latin America, Europe, and Middle East & Africa
Company Share, Market Analysis and Size, Competitive Landscape, Growth Factors, and Trends, and Revenue Forecast
Key Players Covered in the Report
Biogen; Novartis AG; F. Hoffman-La Roche Ltd.; Astellas Pharma Inc.; Pfizer, Inc.; Genzyme Corporation; Isis Pharmaceuticals, Inc.; Boehringer Ingelheim GmbH; Regeneron Pharmaceuticals, Inc.; Ionis Pharmaceuticals, Inc.; and Cytokinetics, Inc.
Market Segment Insights
Intrathecal segment is projected to hold a key market share
On the basis of routes of administration, the spinal muscular atrophy treatment market is bifurcated into oral and intrathecal. The intrathecal segment is expected to hold a key share of the market in the coming years. Spinraza is administered intrathecally. RG 7916 and RG 6083 (Olesoxime) are treatment therapies that are projected to be commercialized. These therapies are administered orally. Hence, oral route of administration is an important route for clinical studies.
Drugs segment is anticipated to expand at a rapid CAGR
Based on treatments, the market is divided into gene therapy and drugs. The drugs segment accounted largest market share and is expected to expand at a rapid CAGR during the forecast period. The drugs segment is further subdivided into Spinraza, RG6083, and RG7916. Till date only approved drug is Spinraza. This drug was approved by the U.S. FDA in December 2016. Spinraza is developed by collaboration of Biogen and Ionis Pharmaceuticals. In 2016, it generated revenue of around 4.5 million.
On the other hand, the gene therapy segment is anticipated to expand at a rapid pace during the forecast period. It is an alternative to drugs. AVXS-101 is only SMA gene therapy and it is being tested by AveXis, Inc. The Orphan Drug Designation and Breakthrough Therapy Designation are used for the treatment of all types of spinal muscular atrophy. To treat SMA type I, Fast Track Designation is used.
Type I segment is expected to expand at a rapid pace
In terms of types, the spinal muscular atrophy treatment market is segmented into type I, type II, type III, and type IV. The type I segment is anticipated to expand at a rapid pace during the forecast period as it is the most common type of SMA. Growing prevalence of type I and increasing focus of companies on novel drug development for SMA treatment are major factors responsible for the segment growth. AveXis, Inc. developed AVXS-10 which is an investigational gene therapy. The rising number of clinical studies of type I disease is key factor propelling the market growth.
North America is expected to dominate the market
On the basis of regions, the spinal muscular atrophy treatment market is categorized as Asia Pacific, North America, Latin America, Europe, and Middle East & Africa. The market of North America is anticipated to dominate the market during the forecast period. The regional market growth can be attributed to the maximum revenue generation in the U.S. market. Moreover, presence of Biogen Inc. in the U.S., increase in the adoption of Spinraza, and high disposable income are anticipated to fuel the regional market growth. Rising number of initiatives taken by government is also projected to drive the market expansion.
However, the market of Asia Pacific is expected to exhibit a rapid growth rate in the coming years owing to the rapidly developing health infrastructure in emerging countries such as China and India. Increase in disposable income is anticipated to drive the market growth over the forecast period.
The global spinal muscular atrophy treatment market has been segmented on the basis of
Routes of Administration
- Gene Therapy
- RG6083 (Olesoxime)
- Type I
- Type II
- Type III
- Type IV
- Asia Pacific
- North America
- Latin America
- Middle East & Africa
- Novartis AG
- F. Hoffman-La Roche Ltd.
- Astellas Pharma Inc.
- Pfizer, Inc.
- Genzyme Corporation
- Isis Pharmaceuticals, Inc.
- Boehringer Ingelheim GmbH
- Regeneron Pharmaceuticals, Inc.
- Ionis Pharmaceuticals, Inc.
- Cytokinetics, Inc.
Key players competing in the spinal muscular atrophy treatment market are Biogen; Novartis AG; F. Hoffman-La Roche Ltd.; Astellas Pharma Inc.; Pfizer, Inc.; Genzyme Corporation; Isis Pharmaceuticals, Inc.; Boehringer Ingelheim GmbH; Regeneron Pharmaceuticals, Inc.; Ionis Pharmaceuticals, Inc.; and Cytokinetics, Inc. Major market players are adopting several business strategies such as mergers, acquisitions, partnerships, collaborations, capacity expansion, and product launches to enhance their market shares.