The growth of the Spinal muscular atrophy (SMA) 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.
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.
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.
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.
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.
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