Italy Batten's Disease (CLN-2) Therapeutics Market Analysis

Italy Batten's Disease (CLN-2) Therapeutics Market Analysis


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Italy Batten?s Disease (CLN-2) Therapeutics market is projected to grow from $xx Mn in 2023 to $xx Mn by 2030, registering a CAGR of xx% during the forecast period of 2023 ? 2030. Neuronal Ceroid Lipofuscinosis (NCL), often known as Batten disease, is a rare and severe autosomal recessive neurodegenerative lysosomal storage condition brought on by cellular buildup of aberrant auto-fluorescent lipoproteins (lipofuscin). Seizures and a progressive loss of movement, verbal, visual, and cognitive abilities are features of this neurodegenerative condition that primarily affects the nerve system. Research advancements in nanotechnology could result in the creation of fresh therapies that would benefit persons who suffer from this ailment. Additionally, it is a paediatric brain condition that is extremely rare yet progresses quickly. As a result, Batten's Disease may be exacerbated by the predicted rising prevalence of disease. Therapeutics market (CLN-2). The market for Batten's Disease (CLN-2) Therapeutics is constrained by a lack of treatment and symptoms that are challenging to identify. Regenxbio, Polaryx Therapeutics and Abeona Therapeutics are some of the key players in the market.

ID: IN10ITRD011 CATEGORY: Rare Diseases GEOGRAPHY: Italy AUTHOR: Sakshi Mantri

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Italy Batten's Disease (CLN-2) Therapeutics Market Analysis Summary

Italy Batten's Disease (CLN-2) Therapeutics Market is valued at around $6 Mn in 2022 and is projected to reach $10.31 Mn by 2030, exhibiting a CAGR of 7% during the forecast period 2023-2030.

Children who have Batten disease (BD), also known as neuronal ceroid lipofuscinosis (NCL), are affected by a rare group of very serious neurodegenerative diseases. It is characterised by the buildup of dangerous compounds called lipofuscins inside cells, notably in the brain, which causes the progressive loss of nerve cells, blindness, and the emergence of neurological issues. One of the most prevalent types of LSDs is late infantile neuronal ceroid lipofuscinosis type 2 (CLN2). CLN2 is produced by the lysosomal enzyme tripeptidyl peptidase 1 (TPP1), a serine protease that localises to lysosomes and extracellularly and cleaves tripeptides off the N-terminus of proteins. Without a functional TPP1, lipofuscin aggregates are not properly cleared, which causes them to gradually build up in lysosomes. Although these aggregates accumulate in the lysosomes of all cells, the central nervous system's (CNS) and retina's neurons suffer the most damage, which manifests as progressive degeneration. Numerous organ systems and tissues are thus impacted, and early loss of neurological and visual abilities causes severe impairment and early death. Therefore, it is crucial that functional TPP1 is delivered to the CNS in an effective manner. Due to the nature of the condition and the dearth of efficient medicines that can access lysosomes in the CNS, treating BD is difficult. The majority of BD treatment focuses on symptom management and supportive care because there is currently no known cure. This could entail taking drugs to prevent seizures, getting occupational and physical therapy to increase function and mobility, and using aids like wheelchairs and feeding tubes. The most advanced method of treating CLN2 is enzyme replacement therapy (ERT), which is already used in clinics. However, only catheters inserted into the lateral ventricle may successfully cure patients (intracerebroventricular). Research advancements in nanotechnology could result in the creation of fresh therapies that would benefit persons who suffer from this ailment. Additionally, it is a paediatric brain condition that is extremely rare yet progresses quickly. Therefore, the market for Batten's Illness (CLN-2) Therapeutics may be driven by the predicted rise in disease prevalence. The market for Batten's Disease (CLN-2) Therapeutics is constrained by a lack of treatment and symptoms that are challenging to identify. Regenxbio, Polaryx Therapeutics and Abeona Therapeutics are some of the key players in the market.

Market dynamics

Market Drivers

The difficulties in treating BD underline the necessity for ongoing research into fresh, efficient treatments for this debilitating condition. Research advancements in nanotechnology could result in the creation of fresh therapies that would benefit persons who suffer from this ailment. Additionally, it is a paediatric brain condition that is extremely rare yet progresses quickly. Therefore, the market for Batten's Illness (CLN-2) Therapeutics may be driven by the predicted rise in disease prevalence.

Market Developments

A recombinant human tripeptidyl peptidase-1 (rhTPP1) is being developed to treat the CLN2 condition, a kind of Batten disease.

At a meeting with the Division of Rare Diseases and Medical Genetics (DRDMG) in mid-April, Theranexus, a biopharmaceutical company pioneering the treatment of neurological diseases, and the Beyond Batten Disease Foundation (BBDF) announced that the FDA had approved the design and primary and secondary endpoints of the pivotal Phase III trial for Batten disease CLN3.

RegenxBio is now in Phase II of the clinical development of RGX-181 for Batten disease.

According to RUSH researchers, the FDA-approved lipid-lowering drug gemfibrozil may stop the progression of juvenile Batten disease.

Restraints in Batten’s Disease (CLN-2) Therapeutics market

  • Difficult to note symptoms

The juvenile brain condition known as CLN2 (late infantile neuronal ceroid lipofuscinosis type 2) is extremely rare and fast developing. Because it is a childhood illness, the patient in this case cannot assist the doctor by pointing out other symptoms. Typically, parents are asymptomatic carriers.

  • Lack of treatment

Although there is currently no specific treatment for Batten disease, there are methods to control the symptoms and improve the child's comfort. The majority of Batten Disease treatments now available target the symptoms of the condition. Anti-convulsants and immunosuppressants are still the mainstays of this disease's therapy, helping to keep patients clinically comfortable and stop seizures and other symptoms of the disease by using medicinal therapies.

Epilepsy or seizures are the symptoms that are most frequently noted. Anti-convulsant medications, such as valproic acid and lamotrigine, alone or in combination with clonazepam, are prescribed by a doctor to manage or lessen seizures. Carbamazepine, oxcarbazepine, and phenytoin are among the more anti-convulsant medications that may be administered.

  • Difficult to diagnose

The most frequent health concern that children between the ages of 2-4 have is seizures. Children frequently develop language delays over time. Both of these symptoms do not directly indicate Batten's disease, making a diagnosis challenging.

Key players

Orchard Therapeutics uniQure Pfizer Alexion Pharmaceuticals Genzyme Sangamo Therapeutics Magenta Therapeutics Ceregene Ionis Pharmaceuticals BioMarin Pharmaceutical

1. Executive Summary
1.1 Disease Overview
1.2 Global Scenario
1.3 Country Overview
1.4 Healthcare Scenario in Country
1.5 Patient Journey
1.6 Health Insurance Coverage in Country
1.7 Active Pharmaceutical Ingredient (API)
1.8 Recent Developments in the Country

2. Market Size and Forecasting
2.1 Epidemiology of Disease
2.2 Market Size (With Excel & Methodology)
2.3 Market Segmentation (Check all Segments in Segmentation Section)

3. Market Dynamics
3.1 Market Drivers
3.2 Market Restraints

4. Competitive Landscape
4.1 Major Market Share

4.2 Key Company Profile (Check all Companies in the Summary Section)

4.2.1 Company
4.2.1.1 Overview
4.2.1.2 Product Applications and Services
4.2.1.3 Recent Developments
4.2.1.4 Partnerships Ecosystem
4.2.1.5 Financials (Based on Availability)

5. Reimbursement Scenario
5.1 Reimbursement Regulation
5.2 Reimbursement Process for Diagnosis
5.3 Reimbursement Process for Treatment

6. Methodology and Scope

Market segmentations for Italy Batten’s Disease (CLN-2) Therapeutics market

By treatment

  • Medicine- Anti-Convulsant
  • Therapy – enzyme replacement therapy, Physical and Occupational Therapy

By Route of administration

  • Oral
  • Injectable
  • Other

By end user

  • Hospital
  • Clinic
  • pharmacy

Methodology for Database Creation

Our database offers a comprehensive list of healthcare centers, meticulously curated to provide detailed information on a wide range of specialties and services. It includes top-tier hospitals, clinics, and diagnostic facilities across 30 countries and 24 specialties, ensuring users can find the healthcare services they need.​

Additionally, we provide a comprehensive list of Key Opinion Leaders (KOLs) based on your requirements. Our curated list captures various crucial aspects of the KOLs, offering more than just general information. Whether you're looking to boost brand awareness, drive engagement, or launch a new product, our extensive list of KOLs ensures you have the right experts by your side. Covering 30 countries and 36 specialties, our database guarantees access to the best KOLs in the healthcare industry, supporting strategic decisions and enhancing your initiatives.

How Do We Get It?

Our database is created and maintained through a combination of secondary and primary research methodologies.

1. Secondary Research

With many years of experience in the healthcare field, we have our own rich proprietary data from various past projects. This historical data serves as the foundation for our database. Our continuous process of gathering data involves:

  • Analyzing historical proprietary data collected from multiple projects.
  • Regularly updating our existing data sets with new findings and trends.
  • Ensuring data consistency and accuracy through rigorous validation processes.

With extensive experience in the field, we have developed a proprietary GenAI-based technology that is uniquely tailored to our organization. This advanced technology enables us to scan a wide array of relevant information sources across the internet. Our data-gathering process includes:

  • Searching through academic conferences, published research, citations, and social media platforms
  • Collecting and compiling diverse data to build a comprehensive and detailed database
  • Continuously updating our database with new information to ensure its relevance and accuracy

2. Primary Research

To complement and validate our secondary data, we engage in primary research through local tie-ups and partnerships. This process involves:

  • Collaborating with local healthcare providers, hospitals, and clinics to gather real-time data.
  • Conducting surveys, interviews, and field studies to collect fresh data directly from the source.
  • Continuously refreshing our database to ensure that the information remains current and reliable.
  • Validating secondary data through cross-referencing with primary data to ensure accuracy and relevance.

Combining Secondary and Primary Research

By integrating both secondary and primary research methodologies, we ensure that our database is comprehensive, accurate, and up-to-date. The combined process involves:

  • Merging historical data from secondary research with real-time data from primary research.
  • Conducting thorough data validation and cleansing to remove inconsistencies and errors.
  • Organizing data into a structured format that is easily accessible and usable for various applications.
  • Continuously monitoring and updating the database to reflect the latest developments and trends in the healthcare field.

Through this meticulous process, we create a final database tailored to each region and domain within the healthcare industry. This approach ensures that our clients receive reliable and relevant data, empowering them to make informed decisions and drive innovation in their respective fields.

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Last updated on: 04 July 2023
Updated by: Dr. Purav Gandhi

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