CDKL5 Deficiency Disorder (CDD) is a rare developmental epileptic encephalopathy (DEE) caused by mutations in the CDKL5 gene.
CDKL5 Deficiency Disorder Emerging Drugs
- Ganaxolone: Marinus Pharmaceuticals
- Soticlestat (TAK935/OV935): Takeda/Ovid Therapeutics
- Fintepla (ZX008): Zogenix
- Translarna (ataluren): PTC Therapeutics
The available therapeutics options in CDKL5 Deficiency Disorder (CDD) treatment Landscape aim to reduce the occurrence of seizures.
The dynamics of CDKL5 Deficiency Disorder (CDKL5 Deficiency Disorder (CDD)) market is anticipated to change in the coming years owing to the improvement in the diagnosis methodologies, raising awareness of the diseases, incremental healthcare spending across the world, and also expects the launch of emerging therapies during the forecast period of 2020–2030.
There are several companies that are also undergoing preclinical and research and developmental program for the treatment of CDD. Few of them are Ultragenyx Pharmaceutical (UX055), Amicus Therapeutics, Abcam, Teruyuki Tanaka, and Trinity College Institute of Neuroscience.
Soticlestat (TAK-935/OV935) is a potent, highly-selective, first-in-class inhibitor of the enzyme cholesterol 24-hydroxylase (CH24H) being investigated as an anti-epileptic drug (AED). Ovid and Takeda believe that its novel mechanism of action may potentially treat rare epilepsies by inhibiting CH24H to decrease 24HC levels, decreasing glutamate hyperactivity effectively. This mechanism of action may be especially important in CDKL5 Deficiency Disorder (CDD) and Dup15q since the NMDA receptor-mediated synaptic transmission underlies the pathological mechanisms of these syndromes. A Phase Ib/IIa study has been completed, and OV935 was shown to reduce the median seizure frequency and was generally safe and well-tolerated. Currently, this molecule is investigated in the Phase II stage of development for the treatment of patients with Cyclin- Dependent Kinase-Like 5 Deficiency Disorder.
Ganaxolone, developed by Marinus Pharmaceuticals, is an allosteric modulator of GABAA receptors acting through binding sites which are distinct from the benzodiazepine binding site. It has activity in a broad range of animal models of epilepsy. In early Phase II studies, Ganaxolone has been shown to have activity in adult patients with partial-onset seizures and epileptic children with a history of infantile spasms. Currently, this molecule is being investigated in Phase III for treatment in children and young adults with Cyclin-dependent Kinase-like 5 (CDKL5) Deficiency Disorder (CDKL5 Deficiency Disorder (CDD)). The drug has received orphan drug designation by the European Union and the US FDA.
Scope of the report
- The Cyclin-Dependent Kinase-Like 5 (CDKL5) Deficiency Disorder report provides an overview of therapeutic pipeline activity and therapeutic assessment of the products by development stage, product type, route of administration, molecule type, and MOA type for Cyclin-Dependent Kinase-Like 5 (CDKL5) Deficiency Disorder across the complete product development cycle, including all clinical and nonclinical stages.
- It comprises of detailed profiles of Cyclin-Dependent Kinase-Like 5 (CDKL5) Deficiency Disorder therapeutic products with key coverage of developmental activities, including technology, collaborations, licensing, mergers and acquisition, funding, designations and other product-related details
- Detailed Cyclin-Dependent Kinase-Like 5 (CDKL5) Deficiency Disorder research and development progress and trial details, results wherever available, are also included in the pipeline study.
- Coverage of dormant and discontinued pipeline projects along with the reasons if available across Cyclin-Dependent Kinase-Like 5 (CDKL5) Deficiency Disorder.
1. Report Introduction
2. Cyclin-Dependent Kinase-Like 5 (CDKL5) Deficiency Disorder
2.1. Overview
2.2. History
2.3. Cyclin-Dependent Kinase-Like 5 (CDKL5) Deficiency Disorder Symptoms
2.4. Causes
2.5.Pathophysiology
2.6. Cyclin-Dependent Kinase-Like 5 (CDKL5) Deficiency Disorder Diagnosis
2.6.1. Diagnostic Guidelines
3. Cyclin-Dependent Kinase-Like 5 (CDKL5) Deficiency Disorder Current Treatment Patterns
3.1. Cyclin-Dependent Kinase-Like 5 (CDKL5) Deficiency Disorder Treatment Guidelines
4. Cyclin-Dependent Kinase-Like 5 (CDKL5) Deficiency Disorder – DelveInsight’s Analytical Perspective
4.1. In-depth Commercial Assessment
4.1.1. Cyclin-Dependent Kinase-Like 5 (CDKL5) Deficiency Disorder companies collaborations, Licensing, Acquisition -Deal Value Trends
4.1.1.1. Assessment Summary
4.1.2. Cyclin-Dependent Kinase-Like 5 (CDKL5) Deficiency Disorder Collaboration Deals
4.1.2.1. Company-Company Collaborations (Licensing / Partnering) Analysis
4.1.2.2. Company-University Collaborations (Licensing / Partnering) Analysis
4.1.2.3. Cyclin-Dependent Kinase-Like 5 (CDKL5) Deficiency Disorder Acquisition Analysis
5. Therapeutic Assessment
5.1. Clinical Assessment of Pipeline Drugs
5.1.1. Assessment by Phase of Development
5.1.2. Assessment by Product Type (Mono / Combination)
5.1.2.1. Assessment by Stage and Product Type
5.1.3. Assessment by Route of Administration
5.1.3.1. Assessment by Stage and Route of Administration
5.1.4. Assessment by Molecule Type
5.1.4.1. Assessment by Stage and Molecule Type
5.1.5. Assessment by MOA
5.1.5.1. Assessment by Stage and MOA
5.1.6. Assessment by Target
5.1.6.1. Assessment by Stage and Target
6. Cyclin-Dependent Kinase-Like 5 (CDKL5) Deficiency Disorder Late Stage Products (Phase-III)
7. Cyclin-Dependent Kinase-Like 5 (CDKL5) Deficiency Disorder Mid Stage Products (Phase-II)
8. Early Stage Products (Phase-I)
9. Pre-clinical Products and Discovery Stage Products
10. Inactive Products
11. Dormant Products
12. Cyclin-Dependent Kinase-Like 5 (CDKL5) Deficiency Disorder Discontinued Products
13. Cyclin-Dependent Kinase-Like 5 (CDKL5) Deficiency Disorder Product Profiles
13.1. Drug Name: Company
13.1.1. Product Description
13.1.1.1. Product Overview
13.1.1.2. Mechanism of action
13.1.2. Research and Development
13.1.2.1. Clinical Studies
13.1.3. Product Development Activities
13.1.3.1. Collaboration
13.1.3.2. Agreements
13.1.3.3. Acquisition
13.1.3.4. Patent Detail
13.1.4. Tabulated Product Summary
13.1.4.1. General Description Table
Detailed information in the report?
14. Cyclin-Dependent Kinase-Like 5 (CDKL5) Deficiency Disorder Key Companies
15. Cyclin-Dependent Kinase-Like 5 (CDKL5) Deficiency Disorder Key Products
16. Dormant and Discontinued Products
16.1. Dormant Products
16.1.1. Reasons for being dormant
16.2. Discontinued Products
16.2.1. Reasons for the discontinuation
17. Cyclin-Dependent Kinase-Like 5 (CDKL5) Deficiency Disorder Unmet Needs
18. Cyclin-Dependent Kinase-Like 5 (CDKL5) Deficiency Disorder Future Perspectives
19. Cyclin-Dependent Kinase-Like 5 (CDKL5) Deficiency Disorder Analyst Review
20. Appendix
21. Report Methodology
21.1. Secondary Research
21.2. Expert Panel Validation
Media Contact
Company Name: DelveInsight
Contact Person: Priya Maurya
Email: Send Email
Phone: +919650213330
City: Albany
State: New York
Country: United States
Website: https://www.delveinsight.com/