Australia Batten’s Disease (CLN-2) Market Analysis


  • Type: Rare Disease Area
  • Published : April 2021
  • Region: APAC
  • Country: Australia
  • Report ID : 4095

  • Format: PPT, PDF

Australian Batten’s Disease (CLN-2) Therapeutics Market: Segmented by Drugs, Treatment, Route of Administration, and Distribution Channel – Growth, Trends, and Forecasts (2020 – 2028)

SKU: 4095 Categories: ,

The Australian Batten’s Disease (CLN-2) 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.

Batten disease (Jansky-Bielschowsky disease) is the common name for a family of severe and progressive neurological disorders (also known as neuronal ceroid lipofuscinoses – NCLs) that primarily affect children. Collectively, Batten disease is the most common inherited pediatric neurodegenerative disorder worldwide and the most common form of childhood dementia. Batten disease is a genetic disease, caused by mutations in ceroid lipofuscinosis neuronal (CLN) genes. The different types of Batten disease are classified according to the defective CLN gene that is causing the disorder (e.g., CLN1, CLN2, CLN3).

BDSRA Australia estimates that at any one time there are approximately 35 people living with a form of Batten disease in Australia, with an average of three to five new cases of Batten disease diagnosed each year in Australia.

Market Growth Drivers & Restraints

The market is expected to increase as the prevalence of neurological disorders rises, biotechnology and pharmaceutical firms engage more in R&D, the demand for new treatment methods grows, and healthcare expenditure rises.

For Batten disease, just one drug has been approved. Treatment is expensive. In rural places, there is a lack of critical services. Physical therapy market growth is being hampered by a scarcity of healthcare professionals.

1. Australian Batten’s Disease (CLN-2) Therapeutics Market Overview………………..
A. Market Size
2. Market Growth Drivers & Restraints…………………………………………………….
A. Increased Prevalence of Neurological Disorders
B. Adoption of New Treatment Methods
C. Increase in Awareness Levels of Rare Diseases
D. High Treatment Costs
3. Major Batten’s Disease Types………………………………………………………..
A. Congenital NCL
B. Infantile NCL
C. Late Infantile NCL
D. Adult NCL
4. Australian Batten’s Disease (CLN-2) Therapeutics Market Segmentation………….
A. By Drugs
I. Cerliponase Alfa
II. Valproic acid
III. Levetiracetam
IV. Others
B. By Treatment
I. Medication
II. Occupational Therapies
III. Physical Therapies
C. By Route of Administration
I. Oral
II. Parenteral
III. Others
D. By Distribution Channel
I. Hospital Pharmacy
II. Retail Pharmacy
III. Online Pharmacy
5. Australian Batten’s Disease (CLN-2) Major Products 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. BioMarin Company overview, Product & Services, Strategies & Financials
B. Amicus Company overview, Product & Services, Strategies & Financials
C. Pfizer 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 Batten’s disease (CLN-2) Therapeutics Market
B. Future Opportunities
10. Conclusion

By Drugs:

On the basis of drugs, the Batten Disease Therapeutics market can be bifurcated into Cerliponase Alfa, Valproic acid, Levetiracetam, and others. Australian Government-subsidised drug treatment is available for eligible patients with a confirmed diagnosis of CLN2 disease. There is one drug currently subsidized through the Life Saving Drugs Program (LSDP) for the treatment of CLN2 disease. The generic name for this drug is Cerliponase Alfa. The trade name for this drug is Brineura. The current therapeutic landscape for Batten Disease predominantly focuses on disease-associated symptoms. On the whole, Anticonvulsants and Immunosuppressants remain the mainstays for the treatment of this disease, to keep the patient clinically comfortable, to prevent seizures and other associated symptoms with the use of medical interventions.

By Treatment:

On the basis of treatment, the Batten Disease Therapeutics market can be segmented into anticonvulsants, physical therapy, and occupational therapy. As yet, no specific treatment is known that can halt or reverse the symptoms of Batten disease or other NCLs. However, seizures can sometimes be reduced or controlled with anticonvulsant drugs, and other medical problems can be treated appropriately as they arise. At the same time, physical and occupational therapy may help patients retain function as long as possible. Some reports have described a slowing of the disease in children with Batten Disease who were treated with vitamins C and E and with diets low in vitamin A. However, these treatments did not prevent the fatal outcome of the disease.

By Routes of administration:

Based on routes of administration, the Batten Disease Therapeutics market is segmented into oral, & parenteral. The parenteral route will be gaining the maximum share in the forecasted period.

By Distribution Channel:

In terms of distribution channels, the Batten Disease Therapeutics market has been segmented into hospital pharmacies, retail pharmacies, and online pharmacies. The hospital pharmacies segment has accounted for the highest market share as the major therapeutics are prescription products. Therefore, hospital pharmacies are further anticipated also to witness growth during the forecast period.

Top Major Players

The Batten Disease Therapeutics Market has limited players and the prominent players in the market have adopted various strategies to garner maximum market share. These include collaboration, product launch, partnership, and acquisition. Major players operating in the market include BioMarin Pharmaceuticals, Amicus Therapeutics, Pfizer Inc., Abeona Therapeutics Inc. 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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