US Rett Syndrome Drugs Market Analysis

US Rett Syndrome Drugs Market Analysis


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US Rett Syndrome Drugs 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. The market for Rett syndrome will grow over time as a result of expanding female population, rising public healthcare programme spending for patient advocacy and rising global healthcare expenditure. Major global players in Rett Syndrome Drugs Market are Merck KGaA (Germany), Eisai Co., Ltd. (Japan), AstraZeneca (U.K.), Sanofi (France), Novartis AG (Switzerland), Abbott (U.S.), F. Hoffmann-La Roche Ltd. (Switzerland), Teva Pharmaceutical Industries Ltd. (Ireland), Pfizer Inc. (U.S.), GlaxoSmithKline plc (U.K.), WOCKHARDT (Mumbai), Novo Nordisk A/S (Denmark), Glenmark Pharmaceuticals Limited (India), Cipla Inc. (U.S.), Bausch Health Companies Inc. (Canada), Otsuka America Pharmaceutical, Inc. (U.S.), Johnson & Johnson Private Limited (U.S.), Takeda Pharmaceutical Company Limited (Japan), Sumitomo Corporation (Japan), Biocon (India)

ID: IN10USRD111 CATEGORY: Rare Diseases GEOGRAPHY: US AUTHOR: Sakshi Mantri

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US Rett Syndrome Drugs Market Analysis Summary

US Rett Syndrome Drugs Market is valued at around $94.5 Mn in 2022 and is projected to reach $177.92 Mn by 2030, exhibiting a CAGR of 8.23% during the forecast period 2023-2030.

Rett syndrome is an uncommon neurological condition that mostly affects women. The disease is characterised by developmental issues, loss of motor skills, and speech impairment. The condition, which is found in children between the ages of 6 and 18 months, is also caused by a mutation in the MECP2 gene on the X chromosome. Rett syndrome can cause a range of problems, from little to major. Rett syndrome is typically characterized by irregular eye movements, slow growth, irritability and agitation, poor language abilities, problems with hand movement, problems with muscular control and coordination, and breathing problems. Despite the fact that Rett syndrome affects many young individuals, many go on to lead happy lives as adults. Since there are only a few recognized treatments for Rett syndrome, the focus of care is on symptom management and improving the quality of life of patients. Occupational, physical, and speech treatments will be helpful for the majority of the affected youngsters. Commonly used drugs for managing symptoms of Rett syndrome are as follows

Antiepileptic medications like valproic acid and clobazam.

Medicines for treating gastrointestinal diseases like laxatives and proton pump inhibitors.

Melatonin and certain sedatives can be used as sleep aids.

Dopamine agonists like bromocriptine or pramipexole improve coordination and movement in individuals.

Nutritional supplements like omega-3 fatty acids improve the overall health and brain function of patients with Rett syndrome.

Trofinetide an oral solution marketed as a daybed for the treatment of Rett syndrome has recently been approved

The market for Rett syndrome will grow over time as a result of expanding female population, rising public healthcare programme spending, and rising global healthcare expenditure. Major global players in Rett Syndrome  Drugs Market are Merck KGaA (Germany), Eisai Co., Ltd. (Japan), AstraZeneca (U.K.), Sanofi (France), Novartis AG (Switzerland), Abbott (U.S.), F. Hoffmann-La Roche Ltd. (Switzerland), Teva Pharmaceutical Industries Ltd. (Ireland), Pfizer Inc. (U.S.), GlaxoSmithKline plc (U.K.), WOCKHARDT (Mumbai), Novo Nordisk A/S (Denmark), Glenmark Pharmaceuticals Limited (India), Cipla Inc. (U.S.), Bausch Health Companies Inc. (Canada), Otsuka America Pharmaceutical, Inc. (U.S.), Johnson & Johnson Private Limited (U.S.), Takeda Pharmaceutical Company Limited (Japan), Sumitomo Corporation (Japan), Biocon (India).

Market Dynamics

Market Drivers

  • ?Increasing prevalence of Rett syndrome

Rett syndrome's rising prevalence will drive industry expansion. The MECP2 gene mutation that causes Rett syndrome. On the long arm of chromosome X, the MECP2 gene produces methyl-CpG binding protein 2. As people grow older, they experience symptoms. Newborns with Rett syndrome grow and develop normally for the first six months before beginning to display symptoms of the condition. Between the ages of 12 and 18 months, typical changes frequently start to occur.

  • Increasing infrastructure investment in the healthcare sector

The rising cost of healthcare, which helps to develop its infrastructure, is another important aspect affecting the market's growth rate for Rett syndrome. Even more will be impacted by market dynamics by various government organisations' initiatives to upgrade the healthcare infrastructure by boosting funding.

 

  • The expansion of the market will be accelerated by an increase in the number of government awareness-raising initiatives and a rise in disposable income. The expansion of the female population and higher investments in women's public health programmes are expected to speed up the market.

Market Restraints

It is anticipated that this disease's consequences will impede market expansion. Particular Rett syndrome characteristics can raise a child's risk of getting sick. For instance, difficulties breathing or swallowing may lead to aspiration pneumonia. This kind of pneumonia happens when liquids like food, saliva, or other liquids enter the lungs rather than the stomach. Lung problems are more common among scoliosis sufferers. If you encounter uncontrolled seizures, your chance of getting sick rises. On the other side, the high cost of therapies, diagnostic techniques, and symptomatic therapy will reduce the market's rate of expansion. The market for patients with Rett syndrome would face difficulties due to a lack of competent workers and inadequate healthcare facilities in developing nations. Furthermore, a lack of knowledge and rigorous government rules would slow down and limit the market's growth rate.

Notable deals in Rett Syndrome drugs Market

In March 2023, FDA approved the first drug for the treatment of Rett syndrome which is Trofitenide an oral solution marketed as a daybed.

Key players

Neuren Pharmaceuticals Limited Anavex Life Sciences Corp. Newron Pharmaceuticals S.p.A. Acadia Pharmaceuticals Inc. GW Pharmaceuticals plc Ovid Therapeutics Inc. Roche Holding AG Takeda Pharmaceutical Company Limited Neurotrope, Inc. Zogenix, Inc.

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 US Rett Syndrome  Drugs Market

By Type:

  • Classic Rett Syndrome
  • Atypical Rett Syndrome

By Drug type:

  • Branded
  • Generic

By Treatment:

  • Medication
  • Anticonvulsants
  • Physical Therapy
  • Occupational Therapy
  • Speech-language Therapy
  • Others

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

  1. Market Overview
  2. Market Growth Drivers & Restraints
  3. Epidemiology of 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

Based on our many years of experience, we believe that these are the parameters that are critical to decision-making for business stakeholders. Our focused approach to developing reports focused on 10 key parameters, enabled us to arrive at the name “Insights10”.

 

Stage I: Market Data Collection

Primary Interviews: We have developed a network of experts, freelancers, and researchers across countries through which we engage with local experts to gather key data points and assumptions about each market. We also engage regularly with some of the best market research agencies such as Atheneum, GuidePoint, GLG, etc. to conduct surveys and interviews, and build intelligence. We have language translators as a part of our team, who between them can cover 30+ languages allowing us to extract better local insights.

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Stage II: Market Data Analysis and Statistical Model

Market Trends: We generally look at macro parameters and micro indicators. The macro parameters include changes in government policies, demand and supply of the market, government intervention programs, and major market share. The micro indicators are GDP growth, market size, market volume, etc. We also understand nuances specific to each country like the US, Canada, India, Germany, etc., and have worked across 60+ countries and hence not only understand global trends but how these differ by country, how payment models, market structure, cultural parameters, etc. differ in each country.

Market Sizing and Analysis: Our expert data analytics team has created various market forecast models by employing the top-down approach i.e. starting with the large overall market and segmenting different areas and the bottom-up approach i.e. starting with population and epidemiology and rolling up based on spend, etc., estimating the size of the market, and distributing among the geographic and/or product segments.

The top-down approach is mainly used for new product forecasting and the bottom-up approach is used for demand estimation of any product for different countries summed up to form the total market. We are able to round off insights and build stronger forecasts because we always do both these methods and triangulate the final numbers.

The study on the market covers the analysis of the leading geographies such as Asia-Pacific, Africa, Europe, Middle East, North America, and  Latin America for the period of 2022 to 2030. The qualitative analysis covers the industry landscape and trends, market opportunities, competitive landscape, and policy and regulatory scenario, and the quantitative analysis covers different market estimates and forecasts.

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We make sure to finalize the numbers in alignment with the market research. Firstly, our internal experts ensure thorough validation and checking to ensure accurate and precise analysis and then validation is also done using a multiple-data analysis model. Two-level validation is done and entails the finalization of the report scope and the way of representation pattern.

 

 

Stage III: Interpretation and Presentation

Analysis & Interpretation: The information gathered is then analyzed and synthesized. The second series of interviews are done if necessary to check and validate. The future opportunities are analyzed by understanding product commercialization and many other factors. It also comprises the analysis of data discrepancies observed across various data sources. Information procured from secondary and primary results is then, interpreted by considering the following parameters: (a partial list)

  • Establishing market drivers and trends
  • Analyzing the regulatory landscape to understand future growth
  • Market Segment based analysis to obtain revenue/volume
  • ​Analyzing current needs and determining penetration to estimate the market

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Data curated from the analysis and interpretation are drawn to portray all in one consolidated report.

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The foreign exchange rates are calculated on the respective dates and for the respective regions covered in the study.

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Last updated on: 04 July 2023
Updated by: Shivam Zalke

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