Cyclin-Dependent Kinase-Like 5 (CDKL5) Deficiency Disorder - Pipeline Insight, 2024

This “Cyclin-Dependent Kinase-Like 5 (CDKL5) Deficiency Disorder- Pipeline Insight, 2024” report provides comprehensive insights about 5+ companies and 5+ pipeline drugs in CDKL5 Deficiency Disorder pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

Geography Covered

• Global coverage

CDKL5 Deficiency Disorder: Understanding

CDKL5 Deficiency Disorder: Overview

CDKL5 deficiency disorder is a rare developmental encephalopathy (DE) caused by pathogenic variants in the gene cyclin-dependent kinase-like 5 (CDKL5). It is a rare X-linked genetic disorder first identified in 2004. The most common symptoms include early-onset, difficult to control seizures and neurodevelopmental impairment that affects cognitive, motor, speech and visual function. The CDKL5 gene is responsible for creating a protein necessary for normal brain development and function. Pathogenic variants in the CDKL5 gene reduce the amount of functional CDKL5 protein or alter its activity in neurons. A shortage (deficiency) of CDKL5 or impairment of its function disrupts brain development. The exact cause of CDKL5 deficiency disorder is not known and most pathogenic variants within the CDKL5 gene occur spontaneously and are not passed down through families. CDKL5 genetic changes or mutations are found in children diagnosed with many other neurologic disorders, including infantile spasms, Lennox-Gastaut Syndrome, Rett Syndrome, West Syndrome, and autism. The diagnosis is initially suspected based on history, symptoms, and physical examination. Moreover, it is confirmed by the molecular genetic testing that can usually be performed by either a blood or saliva sample that is sent to a laboratory for genetic sequencing. Seizures in CDKL5 deficiency disorder are difficult to treat and often require multiple anti-seizure medications. Further, in many cases, the physical symptoms of CDKL5 can be alleviated and managed with Physiotherapy/physical therapy, occupational therapy, and speech and augmentative communication therapy. Recently in March 2022, the US Food and Developmental Administration (FDA) approved Ztalmy (ganaxolone) to treat seizures associated with CDKL5 deficiency disorder in patients 2 years of age and older. This is the first treatment for seizures associated with CDD and the first treatment specifically for CDKL5 deficiency disorder.

CDKL5 Deficiency Disorder- Pipeline Insight, 2024 report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the CDKL5 Deficiency Disorder pipeline landscape is provided which includes the disease overview and CDKL5 Deficiency Disorder treatment guidelines. The assessment part of the report embraces, in depth CDKL5 Deficiency Disorder commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, CDKL5 Deficiency Disorder collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights

The companies and academics are working to assess challenges and seek opportunities that could influence CDKL5 Deficiency Disorder R&D. The therapies under development are focused on novel approaches to treat/improve CDKL5 Deficiency Disorder.

CDKL5 Deficiency Disorder Emerging Drugs Chapters

This segment of the CDKL5 Deficiency Disorder report encloses its detailed analysis of various drugs in different stages of clinical development, including phase III, II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

CDKL5 Deficiency Disorder Emerging Drugs

Fenfluramine: UCB S.A.Fenfluramine is an investigational serotonin releasing agent that has shown to stimulate multiple 5-HT receptor sub-types through the release of serotonin. Fenfluramine may reduce seizures by acting as an agonist at specific serotonin receptors in the brain, including the 5-HT1D, 5-HT2A, and 5-HT2C receptors, and also by acting on the sigma-1 receptor. Currently, the drug is in Phase III stage of Clinical trial evaluation for the treatment of CDKL5 Deficiency Disorder.

CDKL5 Deficiency Disorder: Therapeutic Assessment

This segment of the report provides insights about the different CDKL5 Deficiency Disorder drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in CDKL5 Deficiency Disorder

There are approx. 5+ key companies which are developing the therapies for CDKL5 Deficiency Disorder. The companies which have their CDKL5 Deficiency Disorder drug candidates in the most advanced stage, i.e. phase III include, UCB S.A.

Phases

This report covers around 5+ products under different phases of clinical development like

• Late stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates

Route of Administration

CDKL5 Deficiency Disorder pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Intra-articular
• Intraocular
• Intrathecal
• Intravenous
• Ophthalmic
• Oral
• Parenteral
• Subcutaneous
• Topical
• Transdermal

Molecule Type

Products have been categorized under various Molecule types such as

• Oligonucleotide
• Peptide
• Small molecule

Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

CDKL5 Deficiency Disorder: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses CDKL5 Deficiency Disorder therapeutic drugs key players involved in developing key drugs.

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging CDKL5 Deficiency Disorder drugs.

CDKL5 Deficiency Disorder Report Insights

• CDKL5 Deficiency Disorder Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

CDKL5 Deficiency Disorder Report Assessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Questions

Current Treatment Scenario and Emerging Therapies:

• How many companies are developing CDKL5 Deficiency Disorder drugs?
• How many CDKL5 Deficiency Disorder drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of CDKL5 Deficiency Disorder?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the CDKL5 Deficiency Disorder therapeutics?
• What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for CDKL5 Deficiency Disorder and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• UCB S.A.
• Ultragenyx Pharmaceutical
• Amicus Therapeutics
• Ovid Therapeutics
• REGENXBIO

Key Products

• Fenfluramine
• UX055
• Soticlestat
• Research Program
• AAV gene therapy

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