UK Spinal Muscular Atrophy (SMA) Market Analysis


  • Type: Rare Disease Area
  • Published : April 2021
  • Region: Europe
  • Country: UK
  • Report ID : 2613

  • Format: PPT, PDF

UK Spinal Muscular Atrophy Therapeutics Market: Segmented by Disease Type, Diagnosis, Therapeutics, and Distribution Channels – Size, Share, Impact, Growth, Trends, and Forecasts (2020 – 2028)

Published Date: June 2021

SKU: 2613 Categories: ,

Report Overview

The UK Spinal Muscular Atrophy Therapeutics market size stood at around USD xx billion in 2020 and is projected to reach USD xx billion by 2028, exhibiting a CAGR of xx% during the forecast period.

Spinal Muscular Atrophy (SMA) is a group of genetic neuromuscular disorders that affect the nerve cells that control voluntary muscles (motor neurons). The loss of motor neurons causes progressive muscle weakness and loss of movement due to muscle wasting (atrophy). The severity of the symptoms, the age at which symptoms, begin, and the genetic cause varies by type. Many types of SMA mainly affect the muscles involved in walking, sitting, arm movement, and head control. Breathing and swallowing may also become difficult as the disease progresses in many types of SMA. In some types of SMA, the loss of motor neurons makes it hard to control the movement of the hands and feet.

SMA types 1, 2, 3, and 4 are caused by changes (or pathogenic mutations) in the SMN1 gene and are inherited in an autosomal recessive manner. The disease generally manifests early in life and is the leading genetic cause of death in infants and toddlers. SMA is caused by defects in the Survival Motor Neuron 1 (SMN1) gene that encodes the SMN protein. The SMN protein is critical to the health and survival of the nerve cells in the spinal cord responsible for muscle contraction (motor neurons).

SMA is one of the most common rare diseases. In the UK approximately 100 children are born with this rare disease each year. Moreover, there are between some 1200 – 2500 children and adults in the UK living with SMA. Approximately one in every 40-60 people is an SMA carrier, which is approximately 1.5 million people in the UK. Approximately one in 6,000 babies born have SMA, and about one in 40 people carry a copy of the altered gene that causes the condition (although they do not have the condition themselves).

Market Growth Drivers

The overall burden of spinal muscular atrophy is one of the highest in terms of cost and healthcare services in rare disorders. The rising incidence of spinal muscular atrophy due to the increasing population is one of the key factors for the growth in the SMA treatment market. This growth is attributed to the rapidly developing health infrastructure in the UK, the initiatives are undertaken by various public, as well as non-profit, organizations for creating awareness regarding SMA, have increased remarkably which is propelling the demand for efficient therapeutic options leading to better patient outcomes.

An increase in R&D investment by major companies will boost the market growth in the forecast period. Granting various drug designation to novel drugs is also accelerating the market growth for spinal muscular atrophy treatment. In recent times, there have been few substantial and very significant product launches in gene therapy for the treatment of spinal muscular atrophy disease.

Market Restraints

However, the high cost of treatment for spinal muscular atrophy is hindering market growth. Additionally, the lack of experienced professionals and less adoption rate of spinal muscular atrophy treatment options is also restraining the market growth.


1. UK Spinal Muscular Atrophy Therapeutics Market Overview……………
A. Market Size
2. Market Growth Drivers and Restraints……………………………………….…
Market Growth Drivers
A. Increase in R&D Investment & Introduction of Novel Therapies
B. Developing Healthcare Infrastructure & Rise in Awareness
Market Restraints
A. Higher Cost of Treatment and Lower Treatment Rates
B. Lack of Experienced Professionals
3. Major Types of Spinal Muscular Atrophy………………………………………….
A. SMA with Progressive Myoclonic Epilepsy (SMA-PME)
B. SMA with Respiratory Distress 1 (SMARD1)
C. Adult-onset Spinal Muscular Atrophy
D. X-linked Infantile Spinal Muscular Atrophy
4. Spinal Muscular Atrophy Therapeutics Market Segmentation…………..
A. By Disease Type
I. Type 1 SMA
II. Type 2 SMA
III. Type 3 SMA
B. Diagnosis
I. Physical Examination
II. Electromyography
III. Muscle Biopsy
IV. Genetic Counselling
V. Chorionic Villus Sampling Test (CVST)
C. By Therapeutics
I. Evrysdi (risdiplam)
II. Spinraza (nusinersen)
III. Zolgensma (onasemnogene abeparvovec)
IV. Physical Therapy
V. Chest Physiotherapy
D. By Distribution Channels
I. Hospital Pharmacies
II. Retail Pharmacies
III. Online Pharmacies
5. Spinal Muscular Atrophy Major Drugs Market Share…………………………
A. Market Analysis, Insights and Forecast – By Revenue Type
6. Competitive Landscape…………………………………………………………………
A. Major Players
B. Products in Pipeline
7. Key Company Profiles………………………………………………………………….
A. Novartis Company overview, Product & Services, Strategies & Financials
B. Biogen Company overview, Product & Services, Strategies & Financials
C. Genentech Company overview, Product & Services, Strategies & Financials
D. Ionis Company overview, Product & Services, Strategies & Financials
8. Healthcare Policies and Regulatory Landscape………………………………
A. Policy changes and Reimbursement scenario
9. Factors Driving Future Growth…………………………………………………
A. New Trends and Development of Spinal Muscular Atrophy Therapeutics Market
B. Future Opportunities
10. Conclusion

Market Segmentation

By Disease Type:

On the basis of disease type, the spinal muscular atrophy treatment market has been segmented into

  • Type 1 SMA
  • Type 2 SMA
  • Type 3 SMA
  • Type 4 SMA

Type 1 SMA generated the highest revenue amongst all the categories in 2020. Thus, type 1 SMA is anticipated to remain the most dominant in terms of revenue as the majority of the cases of the disorder are of this type, which has led to it dominating in terms of market share. Increasing awareness of the Type 2 SMA and increasing adoption of treatment is also anticipated to drive the growth of this type over the forecast period.

By Diagnosis:

On the basis of diagnosis, the spinal muscular atrophy treatment market can be classified into

  • Physical examination
  • Electromyography
  • Muscle Biopsy
  • Genetic Counselling
  • Chorionic villus sampling (CVS)
  • Amniocentesis
  • Genetic Blood Test
  • Others

A physical examination may be done to look for signs of SMA or similar conditions. Electromyography and muscle biopsy have also been used. Genetic counseling helps you to understand that if you’re at risk of having a child with SMA. While chorionic villus sampling (CVS), and Amniocentesis are usually carrying out during pregnancy. If the child has typical symptoms of SMA, a genetic blood test can be done to confirm the condition.

By Therapeutics:

Based on the therapeutics, the spinal muscular atrophy treatment market can be bifurcated into

  • Evrysdi (risdiplam)
  • Spinraza (nusinersen)
  • Zolgensma (onasemnogene abeparvovec)
  • Physical Therapy
  • Chest Physiotherapy
  • Nutritional therapy
  • Use of Ventilators

Nusinersen has been accounted for the maximum share in 2020, which is attributable to the fact that Nusinersen is the most effective drug among others in the market and possesses Orphan market exclusivity expiry till 1 Jun 2029. However, Zolgensma (Onasemnogene Abeparvovec) who gained approval later after Spinraza (which costs £450,000 for the first year and £225,000 for subsequent years), however expected to witness growth in the coming years owing to the rising incidence of spinal muscular atrophy disease and the greater patient adoption rate but this very costly innovative gene therapy with a reported list price of £1.79 million ($2.48 million) per dose, might limit its application in the UK, NHS England said in a statement.

By Distribution Channels:

In terms of distribution channel, the spinal muscular atrophy treatment market can be categorized into

  • Hospital pharmacies
  • Retail pharmacies
  • Online pharmacies

In terms of value, the hospital pharmacies accounted for the leading market share in 2020, which is attributable to the fact that the majority of the medications for spinal muscular atrophy treatment can be only administered intravenously and can so only be done by trained medical professionals. Hence, the hospital pharmacies segment is anticipated to enhance the market share over the forecast period.

Top Major Players

The UK spinal muscular atrophy treatment market is a highly competitive market and major key players are adopting strategies; organic as well as inorganic market strategies. Evrysdi is the only oral-approved drug with the reported list price of £7,900 per 60 mg vial for people with types 1, 2, and 3 SMA while other prominent drugs are administered by the parenteral route and thus possess better efficacy. The prominent key players prevailing in the UK market are Biogen Idec Research Limited, Genentech-Roche Pharmaceuticals Inc, Novartis UK Limited, and Ionis Pharmaceuticals among others.

Research Methodology

Insights10 will provide you reports within 10 key parameters which are:

  1. Market overview
  2. Market growth drivers & restraints
  3. Epidemiology or disease type
  4. Market segmentation
  5. Market share
  6. Competitive landscape
  7. Key company profiles
  8. Healthcare policies & regulatory framework
  9. Reimbursement scenario
  10. Factors driving future growth

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