UK Oculopharyngeal Muscular Dystrophy (OPMD) Market Analysis


  • Type: Rare Disease Area
  • Published : April 2021
  • Region: Europe
  • Country: UK
  • Report ID : 2591

  • Format: PPT, PDF

UK Oculopharyngeal Muscular Dystrophy (OPMD) Therapeutics Market: Segmented by Diagnosis, Therapeutics, and Route of Administration – Size, Share, Impact, Growth, Trends, and Forecasts (2020 – 2028)

Published Date: June 2021


SKU: 2591 Categories: ,

Report Overview

The UK Oculopharyngeal Muscular Dystrophy (OPMD) 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.

Oculopharyngeal Muscular Dystrophy (OPMD) is a rare, adult-onset, progressive myopathy characterized by progressive eyelid ptosis, ophthalmoplegia, dysphagia, dysarthria, and proximal limb weakness. OPMD is a genetic muscle disorder that mainly affects the muscle of the upper eyelids and throat. OPMD is caused by an expansion in the polyalanine tract in the PABPN1 gene (14q11.2), which leads to overexpression of a mutant protein, polyadenylate-binding protein 2, and consequently to the accumulation of nuclear aggregates in the muscles.

Disease onset occurs in the fifth to sixth decade of life. Early signs include ptosis, ophthalmoplegia without diplopia, limb weakness, dysarthria, and dysphagia. Symptoms usually begin after the age of 45 years, and ptosis is the most common presenting feature. In several cases, limb weakness has preceded dysphagia. In 5-10% of patients, the disease is more severe, with ptosis and dysphagia presenting before the age of 45 and incapacitating distal leg weakness occurring before the age of 60. The manifestations of autosomal recessive OPMD usually present later (after the age of 60) than those of the autosomal dominant form.

OPMD is a rare disorder that affects males and females in equal numbers. The OPMD is among the 30 inherited diseases declared by WHO and its results in the weakening and worsening of muscles. OPMD affects less than 5 in 10,000 people in the EU. According to a study, in Europe, the estimated prevalence of oculopharyngeal muscular dystrophy is 1 in 100,000 people. The analysis demonstrates the phenotypic and genotypic characteristics of 31 patients with OPMD in the UK. Ptosis was the first reported symptom in two-thirds of the patients, and half of the subjects studied had evidence of ophthalmoplegia. The PABP2 analysis is a reliable non-invasive diagnostic test for OPMD in the UK population.

Market Growth Drivers

The major driving factor involved in the OPMD therapeutics market is the increased incidence and prevalence of oculopharyngeal muscular dystrophy in the middle-aged population. Conventional therapy mainly focused on symptomatic treatment of Oculopharyngeal Muscular Dystrophy and having lots of off-target effects, this factor providing the additional opportunity for key players present in the OPMD treatment market to expand their research and development activities and expected to drive the growth of oculopharyngeal muscular dystrophy treatment market during the forecast period.

Additionally, the introduction of new disease-modifying and novel drug therapies, increased drug development activities are expected to drive the oculopharyngeal muscular dystrophy treatment market. Furthermore, gene therapies such as mutation suppression, exon skipping are also anticipated to push the OPMD treatment market during the forecast period.

Market Restraints

On the contrary, the high cost of the novel therapies and lack of awareness among individuals are some factors that are expected to hamper the growth of the oculopharyngeal muscular dystrophy treatment market during the forecast period.


1. UK Oculopharyngeal Muscular Dystrophy Therapeutics Market Overview………………………………
A. Market Size
2. Market Growth Drivers and Restraints……………………………………….…
Market Growth Drivers
A. Increasing Incidence and Prevalence in Middle-aged Population
B. Expansion of Research and Development Activities fosters Market Growth
C. Increasing Adoption of Novel Therapeutics & Technological Advancements
Market Restraints
A. Huge Treatment Costs
B. Lack of Awareness
3. Major Types of Oculopharyngeal Muscular Dystrophy………………………………..
A. Autosomal Dominant Oculopharyngeal Muscular Dystrophy
B. Autosomal Recessive Oculopharyngeal Muscular Dystrophy
4. Paroxysmal Nocturnal Haemoglobinuria Therapeutics Market Segmentation…………………….
A. By Diagnostics
I. Blood Tests
II. Enzyme Assay
III. Genetic Testing
IV. Electromyography
V. Biopsy
VI. Others
B. By Therapeutics
I. Non-steroidal Anti-Inflammatory Drugs (NSAIDs)
II. Corticosteroids
III. ACE Inhibitors
IV. CRISPR/Cas9 gene Therapy
V. Gene Replacement Therapy
VI. Molecular Therapy
VII. Stem Cell Research
VIII. Mutation Suppression
IX. Exon Skipping
X. Others
C. By Route of Administration
I. Oral
II. Intravenous (IV)
III. Others
5. Oculopharyngeal Muscular Dystrophy 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. Bioblast 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 OPMD Therapeutics Market
B. Future Opportunities
10. Conclusion

Market Segmentation

By Diagnosis:

Based on the diagnosis, the OPMD therapeutics market can be bifurcated into

  • Blood tests
  • Enzyme assay
  • Genetic testing
  • Electromyography
  • Biopsy
  • Others

The diagnosis of OPMD can be confirmed through commercially available blood tests that detect the specific genetic abnormality in the PABPN1 gene, known as a repeat expansion, that is associated with OPMD (i.e., mutation of the PABPN1 gene).

By Therapeutics:

Based on therapeutics, the OPMD therapeutics market can be segmented into

  • Non-steroidal anti-inflammatory drugs (NSAIDs)
  • Corticosteroids
  • ACE inhibitors
  • Gene Replacement Therapy
  • Molecular Therapy
  • Stem Cell Research
  • Mutation Suppression
  • Exon Skipping
  • Others

Corticosteroids, such as prednisone and deflazacort (Emflaza), which can help muscle strength and delay the progression of OPMD are commonly preferred. Additionally, novel gene therapies such as CRISPR /Cas9, RNA Replacement strategy to develop promising therapy for Oculopharyngeal Muscular Dystrophy.

By Route of Administration:

Based on the route of administration, the OPMD therapeutics market can be segmented into

  • Oral
  • Intravenous (IV)
  • Others

As per the latest report, there is currently only one treatment for people with Oculopharyngeal Muscular Dystrophy available on the UK’s National Health Service (NHS); Cabaletta (trehalose), which is delivered intravenously once a week. Therefore, the intravenous type of the route of administration segment is anticipated to account for the majority of the revenue share during the forecast period.

Top Major Players

Increased demand for efficacious therapies among patients, rising investment in research and development activities, and introduction of novel disease therapies like RNA replacement strategy, and mutation suppression are anticipated to make the OPMD therapeutics market highly competitive. As of now, ‘Seelos Therapeutics’ emerged as a major key player involved in the Oculopharyngeal Muscular Dystrophy treatment market, had agreed with Bioblast Pharma Ltd., to acquire all development and commercial rights to Bioblast’s proprietary drug named Cabaletta (trehalose).

Research Methodology

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  6. Competitive landscape
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  8. Healthcare policies & regulatory framework
  9. Reimbursement scenario
  10. Factors driving future growth

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