Congenital Adrenal Hyperplasia - Pipeline Insight, 2024

This “Congenital Adrenal Hyperplasia- Pipeline Insight, 2024” report provides comprehensive insights about 5+ companies and 8+ pipeline drugs in Congenital Adrenal Hyperplasia 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

Congenital Adrenal Hyperplasia: Understanding

Congenital Adrenal Hyperplasia: Overview

Congenital Adrenal Hyperplasia (CAH) is a group of genetic disorders affecting the adrenal glands, leading to impaired production of cortisol and aldosterone. The most common form, 21-hydroxylase deficiency, results in excess androgen production, causing ambiguous genitalia in females and early puberty in both genders. CAH can cause severe dehydration and salt loss in the classic form, requiring lifelong management with hormone replacement therapy.

Early diagnosis and treatment are crucial for preventing serious complications and improving quality of life. Signs and Symptoms of Congenital Adrenal Hyperplasia (CAH) include ambiguous genitalia in newborn girls, fertility problems in both men and women, rapid growth during childhood but shorter than average adult height, severe acne and excessive body hair (hirsutism) in females, irregular menstrual periods in females and salt-wasting crisis severe dehydration, low blood pressure, low sodium, and high potassium levels.

Classic CAH: The most common form, which is severe and typically presents in infancy or early childhood. Involves a buildup of 11-deoxycortisol and 11-deoxycorticosterone, leading to symptoms similar to those of 21-hydroxylase deficiency but with different hormonal imbalances and Non-Classic CAH: A milder form that may not present until later in childhood or adulthood, with symptoms such as early puberty, acne, and excess body hair. Certain factors may increase your risk of developing Congenital Adrenal Hyperplasia. These include: Genetic Inheritance CAH is inherited in an autosomal recessive pattern, meaning a child must inherit two copies of the mutated gene (one from each parent) to develop the condition.

Family History: Having parents or siblings with CAH increases the risk of inheriting the disorder. Ethnicity: Certain ethnic groups, such as Ashkenazi Jews, have a higher prevalence of specific genetic mutations that cause CAH. Consanguinity Marriages between closely related individuals (such as cousins) increase the likelihood of inheriting the same genetic mutations, raising the risk of CAH.

Diagnosis of Congenital Adrenal Hyperplasia (CAH) typically involves newborn screening, which measures hormone levels such as 17-hydroxyprogesterone (17-OHP) in a blood sample. Elevated levels of 17-OHP indicate CAH. If abnormal results are found, further confirmatory tests, including genetic testing, hormone assays, and ACTH stimulation tests, are conducted. Prenatal diagnosis can be performed through chorionic villus sampling or amniocentesis if there is a known family history of CAH. Early diagnosis is crucial for timely treatment and management to prevent complications.

Treatment for Congenital Adrenal Hyperplasia (CAH) focuses on hormone replacement therapy to correct the hormone imbalances caused by the disorder. Glucocorticoids, such as hydrocortisone or prednisone, are used to replace cortisol and suppress excess androgen production. Mineralocorticoids, like fludrocortisone, are given to replace aldosterone in salt-wasting forms of CAH. Regular monitoring and dose adjustments are essential to manage growth, development, and metabolic needs.

In severe cases, particularly for females with ambiguous genitalia, surgical interventions may be considered. Lifelong medical follow-up is necessary to ensure optimal health outcomes. Patients with CAH often require lifelong medical care and regular monitoring to ensure hormone levels are balanced and to mitigate potential long-term effects on growth, development, and overall health.

"Congenital Adrenal Hyperplasia- Pipeline Insight, 2024" report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Congenital Adrenal Hyperplasia pipeline landscape is provided which includes the disease overview and Congenital Adrenal Hyperplasia treatment guidelines.

The assessment part of the report embraces, in depth Congenital Adrenal Hyperplasia 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, Congenital Adrenal Hyperplasia 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 Congenital Adrenal Hyperplasia R&D. The therapies under development are focused on novel approaches to treat/improve Congenital Adrenal Hyperplasia.

Congenital Adrenal Hyperplasia Emerging Drugs Chapters

This segment of the Congenital Adrenal Hyperplasia report encloses its detailed analysis of various drugs in different stages of clinical development, including phase III, II, II/III 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.

Congenital Adrenal Hyperplasia Emerging Drugs

Crinecerfont: Neurocrine Biosciences, Inc

Crinecerfont is an investigational, oral, selective corticotropin-releasing factor type 1 receptor (CRF1) antagonist being developed to reduce and control excess adrenocorticotropic hormone (ACTH) and adrenal androgens through a glucocorticoid-independent mechanism for the treatment of congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency.

Antagonism of CRF1 receptors in the pituitary has been shown to decrease ACTH levels, which in turn decreases the production of adrenal androgens and potentially the symptoms associated with CAH. In July 2024, U.S. FDA accepted the NDA application for Crinecerfont for pediatric and adult patients with CAH. Currently, the drug is registered for the treatment of Congenital Adrenal Hyperplasia.

Congenital Adrenal Hyperplasia: Therapeutic Assessment

This segment of the report provides insights about the different Congenital Adrenal Hyperplasia drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Congenital Adrenal Hyperplasia

There are approx. 5+ key companies which are developing the therapies for Congenital Adrenal Hyperplasia. The companies which have their Congenital Adrenal Hyperplasia drug candidates in the most advanced stage, i.e. Phase III include, Neurocrine Biosciences, Inc.

Phases

The report covers around 8+ 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

Congenital Adrenal Hyperplasia pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Intravenous
• Subcutaneous
• Oral
• Intramuscular
• Molecule Type

Products have been categorized under various Molecule types such as

• Monoclonal antibody
• Small molecule
• Peptide
• Product Type

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

Congenital Adrenal Hyperplasia: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Congenital Adrenal Hyperplasia 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 Congenital Adrenal Hyperplasia drugs.

Congenital Adrenal Hyperplasia Report Insights

• Congenital Adrenal Hyperplasia Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Congenital Adrenal Hyperplasia 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 Congenital Adrenal Hyperplasia drugs?
• How many Congenital Adrenal Hyperplasia drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Congenital Adrenal Hyperplasia?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Congenital Adrenal Hyperplasia 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 Congenital Adrenal Hyperplasia and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Neurocrine Biosciences, Inc
• Spruce Biosciences
• Lundbeck
• HBM Alpha Therapeutics

Key Products

• Crinecerfont
• Tildacerfont
• Lu AG13909
• HAT 001

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