Cyclin-Dependent Kinase-Like 5 (CDKL5) Deficiency Disorder - Market Insight, Epidemiology and Market Forecast - 2034

Executive Summary

Country Wise-CDD Epidemiology

The epidemiology segment also provides the CDD epidemiology data and findings across the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan.

CDKL5 Deficiency Disorder Drug Chapters

This segment of the CDD report encloses the detailed analysis of the mid- and late-stage (Phase-III and Phase-II) pipeline drugs. It also helps to understand the clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details of each included drug and the latest news and press releases.

CDKL5 Deficiency Disorder Emerging Drugs

TAK935/OV935 (soticlestat): Takeda/Ovid Therapeutics

TAK935/OV935 (soticlestat) is undergoing phase II clinical development trial, which is being developed by Takeda in collaboration with Ovid Therapeutics for the treatment of CDD. OV935/TAK-935 is a potent, highly-selective, first-in-class inhibitor of the enzyme cholesterol 24-hydroxylase (CH24H) being investigated as an antiepileptic drug (AED). CH24H is predominantly expressed in the brain, where it plays a central role in cholesterol homeostasis. CH24H converts cholesterol to 24-hydroxycholesterol (24HC), which then exits the brain into the blood plasma circulation.

Ganaxolone: Marinus Pharmaceuticals

Marinus Pharmaceuticals is developing Ganaxolone, which is in phase III for the treatment of CDD. It is a synthetic analog of allopregnanolone, an endogenous neurosteroid produced in the central nervous system that modulates the brain neurotransmitter GABA. It is an allosteric modulator of GABAA receptors acting through binding sites which are distinct from the benzodiazepine binding site. The drug is currently being developed in three different dose forms (IV, capsule, and liquid) intended to maximize therapeutic reach to adult and pediatric patient populations in both acute and chronic care settings. The drug has also received orphan drug designation by the EU and the USFDA.

Products detail in the report.

CDKL5 Deficiency Disorder Market Outlook

Currently, there are no approved therapies for CDD. The treatment landscape of CDD includes multidisciplinary care, including antiepileptic drugs, anticonvulsants, physiotherapy, occupational therapy, neurological speech therapy, and dietetics. Therapeutic methods in patients with CDD are based on symptomatic drug use to control the most problematic complaints that increase disability and to increase the possibility for the development of the individual patient. A multidisciplinary team approach is the most effective way to deliver necessary treatments aimed at maximizing the individual’s abilities and facilitating any skills that may be emerging. Emphasis should be placed on early intervention therapies such as physical therapy, occupational therapy, and speech and augmentative communication therapy. Important aspects of management include psychosocial support for the family, development of an appropriate education plan, and assessment of available community resources.

Seizures are a major problem for patients with CDD because they are usually severe and difficult to control with medication. No one anti-seizure medication has been found to be uniformly effective to treat CDD. A combination of anti-seizure medications may offer periods of seizure‐freedom lasting anywhere from a few weeks to a few years. Antiepileptic and anticonvulsants medications are given as the first-line treatment to the patients one by one, and if the seizures relapse in such a situation, second-line medications are preferred.

The CDD market outlook of the report helps to build a detailed comprehension of the historic, current and forecasted CDD market trends by analyzing the impact of current therapies on the market, unmet needs, drivers and barriers, and demand of better technology.

This segment gives a thorough detail of CDD market trend of each marketed drug and late-stage pipeline therapy by evaluating their impact based on the annual cost of therapy, inclusion and exclusion criteria’s, mechanism of action, compliance rate, growing need of the market, increasing patient pool, covered patient segment, expected launch year, competition with other therapies, brand value, their impact on the market and view of the key opinion leaders. The calculated market data are presented with relevant tables and graphs to give a clear view of the market at first sight.

According to the publisher, CDD 7MM market is expected to change in the study period 2017-2030.

Key Findings

This section includes a glimpse of the CDD 7 MM market.

• The market size of CDD the seven major markets is USD 5.95 Million in 2017.
• According to the publisher estimates, the United States accounts for the highest market size of CDD, in comparison to Japan and EU5 (the United Kingdom, Germany, Italy, France, and Spain).
• Among the EU5 countries, Germany had the highest market size in 2017.
• Among 7MM, Japan accounts for the third-highest market size during the forecast period 2017-2030, at a CAGR of 27.37%, which is expected to grow due to launch of emerging therapies.

Market Outlook for Seven Major Markets

This section provides the total CDD market size and market size by therapies in the United States, Germany, France, Italy, Spain, the United Kingdom and Japan.

CDKL5 Deficiency Disorder Drugs Uptake

This section focusses on the rate of uptake of the potential drugs that are expected to get launched in the market during the study period 2017-2030. The analysis covers CDD market uptake by drugs; patient uptake by therapies; and sales of each drug.

This helps in understanding the drugs with the most rapid uptake, reasons behind the maximal use of new drugs and allow the comparison of the drugs on the basis of market share and size which again will be useful in investigating factors important in market uptake and in making financial and regulatory decisions.

CDKL5 Deficiency Disorder Development Activities

The report provides insights into different therapeutic candidates in phase II, and phase III stage. It also analyzes key players involved in developing targeted therapeutics.

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing and patent details for CDD emerging therapies.

Competitive Intelligence Analysis

The publisher performs competitive and market intelligence analysis of the CDD market by using various competitive intelligence tools that include-SWOT analysis, PESTLE analysis, Porter’s five forces, BCG Matrix, Market entry strategies, etc. The inclusion of the analysis entirely depends upon the data availability.

Scope of the Report

• The report covers the descriptive overview of CDD, explaining its causes, signs and symptoms, pathogenesis and currently available therapies.
• Comprehensive insight has been provided into the CDD epidemiology and treatment.
• Additionally, an all-inclusive account of both the current and emerging therapies for CDD is provided, along with the assessment of new therapies, which will have an impact on the current treatment landscape.
• A detailed review of CDD market; historical and forecasted is included in the report, covering the 7MM drug outreach.
• The report provides an edge while developing business strategies by understanding trends shaping and driving the 7MM CDD market.

Report Highlights

• CDD market is anticipated to increase at a CAGR of 26.68% for the study period 2017-2030.
• The United States accounted for more than half (53.60%) of the market share in the 7MM.
• Ganaxolone is projected to lead the market with the largest market share in 2030.
• In the coming years, CDD market is set to change due to the rising awareness of the disease, and incremental healthcare spending across the world; which would expand the size of the market to enable the drug manufacturers to penetrate more into the market.
• The companies and academics are working to assess challenges and seek opportunities that could influence CDD R&D. The therapies under development are focused on novel approaches to treat/improve the disease condition.
• Major players are involved in developing therapies for CDD. Launch of emerging therapies will significantly impact the CDD market.
• Our in-depth analysis of the pipeline assets across different stages of development (phase III and phase II), different emerging trends and comparative analysis of pipeline products with detailed clinical profiles, key competitors, launch date along with product development activities will support the clients in the decision-making process regarding their therapeutic portfolio by identifying the overall scenario of the research and development activities.

CDD Report Insights

• Patient Population
• Therapeutic Approaches
• Sciatica Pipeline Analysis
• Sciatica Market Size and Trends
• Market Opportunities
• Impact of upcoming Therapies

CDD Report Key Strengths

• Eleven Years Forecast
• 7MM Coverage
• CDD Epidemiology Segmentation
• Key Cross Competition
• Highly Analyzed Market
• Drugs Uptake

CDD Report Assessment

• Current Treatment Practices
• Unmet Needs
• Pipeline Product Profiles
• Market Attractiveness
• Market Drivers and Barriers

Companies and Products

• Marinus Pharmaceuticals: Ganaxolone
• Ovid Therapeutics/Takeda: Soticlestat
• Zogenix: Fintepla (ZX008)
• PTC Therapeutics: Translarna (ataluren) 

Key Questions Answered

Market Insights:

• What was the CDD market share (%) distribution in 2017 and how it would look like in 2030?
• What would be the CDD total market size as well as market size by therapies across the 7MM during the forecast period (2020-2030)?
• What are the key findings pertaining to the market across the 7MM and which country will have the largest CDD market size during the forecast period (2020-2030)?
• At what CAGR, the CDD market is expected to grow at the 7MM level during the forecast period (2020-2030)?
• What would be the CDD market outlook across the 7MM during the forecast period (2020-2030)?
• What would be the CDD market growth until 2030, and what will be the resultant market size in the year 2030?
• How would the market drivers, barriers and future opportunities affect the market dynamics and subsequent analysis of the associated trends?

Epidemiology Insights:

• What is the disease risk, burden and unmet needs of CDD?
• What is the historical CDD patient pool in the United States, EU5 (Germany, France, Italy, Spain, and the UK) and Japan?
• What would be the forecasted patient pool of CDD at the 7MM level?
• What will be the growth opportunities across the 7MM with respect to the patient population pertaining to aspergillosis?
• Out of the countries mentioned above, which country would have the highest prevalent population of CDD during the forecast period (2020-2030)?
• At what CAGR the population is expected to grow across the 7MM during the forecast period (2020-2030)?

Current Treatment Scenario, Marketed Drugs and Emerging Therapies:

• What are the current options for the treatment of CDD along with the approved therapy?
• What are the current treatment guidelines for the treatment of CDD in the US and Europe?
• What are the CDD marketed drugs and their MOA, regulatory milestones, product development activities, advantages, disadvantages, safety and efficacy, etc.?
• How many companies are developing therapies for the treatment of CDD?
• How many therapies are developed by each company for the treatment of CDD?
• How many emerging therapies are in the mid-stage and late stage of development for the treatment of CDD?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the CDD therapies?
• What are the recent novel therapies, targets, mechanisms of action and technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for CDD and their status?
• What are the key designations that have been granted for the emerging therapies for CDD?
• What are the 7MM historical and forecasted market of CDD?

Reasons to Buy

• The report will help in developing business strategies by understanding trends shaping and driving the CDD.
• To understand the future market competition in the aspergillosis market and Insightful review of the key market drivers and barriers.
• Organize sales and marketing efforts by identifying the best opportunities for CDD in the US, Europe (Germany, Spain, Italy, France, and the United Kingdom) and Japan.
• Identification of strong upcoming players in the market will help in devising strategies that will help in getting ahead of competitors.
• Organize sales and marketing efforts by identifying the best opportunities for CDD market.
• To understand the future market competition in the CDD market.

• Marinus Pharmaceuticals
• Ovid Therapeutics/Takeda
• Zogenix
• PTC Therapeutics