The ‘CDKL5 Deficiency Disorder (CDD) - Market Insights, Epidemiology and Market Forecast-2030’ report delivers an in-depth understanding of the CDD, historical and forecasted epidemiology as well as the CDD market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan.
The CDD market report provides current treatment practices, emerging drugs, and market share of the individual therapies, current and forecasted 7MM CDD market size from 2017 to 2030. The report also covers current CDD treatment practice/algorithm, market drivers, market barriers and unmet medical needs to curate the best of the opportunities and assesses the underlying potential of the market.
- The United States
- EU5 (Germany, France, Italy, Spain, and the United Kingdom)
Study Period: 2017-2030
CDKL5 Deficiency Disorder (CDD) Disease Understanding and Treatment Algorithm
CDKL5 Deficiency Disorder (CDD) Overview
CDKL5 deficiency disorder (CDD) is a rare developmental epileptic encephalopathy (DEE), X-linked genetic condition which results in early-onset, difficult to control seizures and severe neuro-developmental impairment. CDKL5 stands for cyclin-dependent kinase-like 5 and mutations in this gene were first identified as disease-causing in 2004. CDD was previously not considered as a separate indication; rather, it was assumed to be an atypical form of Rett Syndrome.
The CDKL5 gene provides instructions for making proteins that are essential for normal brain and neuron development. The condition was previously known as serine/threonine-protein kinase 9 (STK9), and it has been classified as DEE because the genetic change causes epileptic activity as well as severe impairment of the development.
The CDKL5 gene is located on the X chromosome. The X chromosome is one of the sex chromosomes; females have two X’s, and males have one X and one Y chromosome. Although many identified patients are males, because of the location of the gene, this disorder mainly affects females. Affected males may have more severe symptoms than females.
CDKL5 Deficiency Disorder Diagnosis
The most prominent feature of the CDKL5 disorder is the early onset of seizures. The onset of seizures can happen within the first hours, days, weeks or months. Children have been diagnosed with CDKL5 with seizures starting as late as 8 months and in some cases, up to the 2 years. Some mothers have described feeling seizures in the prenatal period.
For infants being seen for the first time, the CDKL5 diagnosis is most often made after a clinical workup for causes of early-life seizures, including genetic testing. For older children, the diagnosis is likely made in the process of revisiting potential causes for epilepsy and neurodevelopmental disorder, using the same kind of genetic testing.
With the introduction of more sophisticated genetic testing, CDKL5 disorder is now being diagnosed in children at a relatively early age. Genetic testing prior to 2009, which sequenced the gene, would not necessarily have picked up deletions. Several individuals subsequently diagnosed with CDKL5 disorder previously were given a negative result. Therefore, a child with the phenotype who has previously tested negative, yet does not have a genetic diagnosis, should be re-tested to include deletion and duplication testing of the CDKL5 gene. The diagnosis is based on a simple blood test. More recently, some labs use a buccal swab of the cheek. The blood or saliva swab is then sent to a special laboratory that performs the genetic test. Recently, CDKL5 testing has become widely available in epilepsy and developmental delay genetic panels, thereby allowing testing of multiple potential disease-causing genes all at the same time at a cost similar to just testing the CDKL5 gene alone.
CDKL5 Deficiency Disorder Treatment
Seizures in CDLK5 disorder are difficult to treat, but it is important to note that some people do well on anticonvulsant monotherapy (one drug at a time). In contrast, others need adjunctive therapy (more than one medication). No single seizure medication has proven to be most effective in controlling seizures in these children. It is relatively typical for a child to require many medication trials. Steroid treatment and the ketogenic diet have been used in the treatment course of some children. Vagus nerve stimulation is also often considered with CDKL5-related seizures. If all other therapies have failed, and a seizure focus can be isolated, surgery may be an option in some situations.
The spectrum of disability and health needs vary widely with people affected by CDKL5 disorder. The conditions are life-shortening due to the complications associated with, but not limited to, the seizure disorder, orthopedic, gastrointestinal issues and autonomic dysfunction. These may require long-term care and attention. Families should work with their primary healthcare physician or specialists to access support services in their community for medically fragile and/or developmentally challenged individuals.
The CDD market report gives a thorough understanding of CDD by including details such as disease definition, causes, mechanism, biomarkers, and diagnosis.
CDKL5 Deficiency Disorder Epidemiology
The disease epidemiology covered in the report provides historical as well as forecasted epidemiology include Prevalence of CDD, Diagnosed Prevalence of CDD, Gender-specific Diagnosed Prevalence of CDD, and clinical characteristics of CDD scenario of CDD in the 7MM covering the United States, EU5 countries (Germany, Spain, Italy, France and the United Kingdom) and Japan from 2017 to 2030.
This section provides glimpses of the CDD epidemiology in the 7MM.
- In the 7MM, the total prevalent population of CDD was estimated to be 16,397 cases in 2017.
- The total diagnosed prevalent population of CDD in the seven major markets was 3,584 in 2017, which is expected to increase in the forecast period
- The United States accounted for the maximum diagnosed prevalent population among the 7MM in 2017
- In EU5 countries, Germany accounted for the highest diagnosed cases of CDD while Spain had the lowest diagnosed prevalent population in 2017
- In 2017, the total diagnosed prevalence of CDD in the United States was 208 in males and 1,315 in females
- There are several types of clinical characteristics of CDD, among which, mainly eight types of characteristics are frequently observed. As per the estimates 1,234, 716, 1,142, 1,218, 1,317, 1,080, 976, 1,295, patients were affected by epileptic spasms, hypsarrhythmia, cortical visual impairment, hand stereotypies, gastrointestinal symptoms, constipation, reflux, and sleep difficulties in 2017.
2 Executive Summary
3 SWOT Analysis
4 CDKL5 Deficiency Disorder (CDD) Market Overview at a Glance
4.1 Market Share (%) Distribution of CDKL5 Deficiency Disorder (CDD) in 2017
4.2 Market Share (%) Distribution of CDKL5 Deficiency Disorder (CDD) in 2030
5 Disease Background and Overview: CDKL5 Deficiency Disorder (CDD)
5.2 Signs and Symptoms of CDD
5.2.1 Focal Seizures
5.2.2 Generalized Seizures
5.3 Inheritance Pattern
5.4 Causes of CDD
5.5 Difference between Rett Syndrome and CDD
5.6 Signaling Mechanism of CDD
5.7 Biomarkers for CDD
5.8 Diagnosis of CDD
5.8.1 Genetic Tests for CDKL5 Variants
5.9 Differential Diagnosis
6 Recognized Establishments
7.1.1 Antiepileptic drugs/medications (AEDs)
7.1.2 Steroid Treatment
7.1.3 Ketogenic Diet
7.1.5 Vagal Nerve Stimulation (VNS)
7.2 Therapeutic Interventions
7.2.1 Physical therapy/Physiotherapy
7.2.2 Occupational Therapy
7.2.3 Neurological Speech Therapy
7.2.4 Music Therapy
7.2.5 Hippotherapy/Equine Therapy
7.2.7 Massage Therapy
7.2.8 Vision Therapy
7.2.9 Health and Social Care
8 Epidemiology and Patient Population
8.1 Key Findings
8.2 Epidemiology Methodology
9 Total Prevalent Population of CDKL5 Deficiency Disorder (CDD) in the 7MM
10 Total Diagnosed Prevalent Population of CDD in the 7MM
11 Epidemiology of CDLK5 Deficiency Disorder (CDD) in the 7MM
11.1.1 Assumptions and Rationale for the seven major markets
11.1 United States
11.1.1 Prevalence of CDD in the United States
11.1.2 Diagnosed Prevalence of CDD in the United States
11.1.3 Gender-specific Diagnosed Prevalence of CDD in the United States
11.1.4 Clinical Characteristics of CDD in the United States
11.2.1 Prevalence of CDD in Germany
11.2.2 Diagnosed Prevalence of CDD in Germany
11.2.3 Gender-specific Diagnosed Prevalence of CDD in Germany
11.2.4 Clinical Characteristics of CDD in Germany
11.3.1 Prevalence of CDD in France
11.3.2 Diagnosed Prevalence of CDD in France
11.3.3 Gender-specific Diagnosed Prevalence of CDD in France
11.3.4 Clinical Characteristics of CDD in France
11.4.1 Prevalence of CDD in Italy
11.4.2 Diagnosed Prevalence of CDD in Italy
11.4.3 Gender-specific Diagnosed Prevalence of CDD in Italy
11.4.4 Clinical Characteristics of CDD in Italy
11.5.1 Prevalence of CDD in Spain
11.5.2 Diagnosed Prevalence of CDD in Spain
11.5.3 Gender-specific Diagnosed Prevalence of CDD in Spain
11.5.4 Clinical Characteristics of CDD Spain
11.6 United Kingdom
11.6.1 Prevalence of CDD in the United Kingdom
11.6.2 Diagnosed Prevalence of CDD in the United Kingdom
11.6.3 Gender-specific Diagnosed Prevalence of CDD in The United Kingdom
11.6.4 Clinical Characteristics of CDD in the United Kingdom
11.7.1 Prevalence of CDD in Japan
11.7.2 Diagnosed Prevalence of CDD in Japan
11.7.3 Gender-specific Diagnosed Prevalence of CDD in Japan
11.7.4 Clinical Characteristics of CDD in Japan
12 Unmet Needs
13 Emerging Drugs
13.1 Key Cross Competition
13.2 Ganaxolone: Marinus Pharmaceuticals
13.2.1 Product Description
13.2.2 Other Developmental Activities
13.2.3 Clinical Development
13.2.4 Product Profile
13.3 Soticlestat (TAK935/OV935): Takeda/Ovid Therapeutics
13.3.1 Product Description
13.3.2 Other Developmental Activities
13.3.3 Clinical Development
13.3.4 Product Profile
13.4 Fintepla (ZX008): Zogenix
13.4.1 Product Description
13.4.2 Clinical Development
13.4.3 Product Profile
13.5 Translarna (ataluren): PTC Therapeutics
13.5.1 Product Description
13.5.2 Clinical Development
13.5.3 Product Profile
13.6 CDKL5 Deficiency Disorder (CDD): Seven Major Market Analysis
13.7 Key Findings
13.8 Market Methodology
14 Attribute Analysis
15 Key Market Assumptions
16 Market Access and Reimbursement
17 CDKL5 Deficiency Disorder (CDD): Seven Major Market Analysis
17.1 Key Findings
17.2 Total Market Size of CDKL5 Deficiency Disorder (CDD) in the 7MM
17.3 Market Size by Therapies in the 7MM
17.4 7MM Market Outlook
17.1 United States Market Size
17.1.1 Total Market size of CDD
17.1.2 Market Size by Therapies
17.2.1 Total Market size of CDD in Germany
17.2.2 Market Size by Therapies
17.3.1 Total Market Size of CDD
17.3.2 Market Size by Therapies
17.4.1 Total Market Size of CDD
17.4.2 Market Size by Therapies
17.5.1 Total Market Size of CDD
17.5.2 Market Size by Therapies
17.6 United Kingdom
17.6.1 Total Market Size of CDD
17.6.2 Market Size by Therapies
17.7 Japan Market Size
17.7.1 Total Market Size of CDD
17.7.2 Market Size by Therapies
18 Case Reports
18.1 Mutation in an alternative transcript of CDKL5 in a boy with early-onset seizures
18.2 CDKL5 Deficiency Disorder: Avery’s Story
19 Market Drivers
20 Market Barriers
22 Report Methodology
23 Publisher Capabilities
25 About the Publisher
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.
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.
- 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
- 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?
- 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.
A selection of companies mentioned in this report includes:
- Marinus Pharmaceuticals
- Ovid Therapeutics/Takeda
- PTC Therapeutics