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Congenital Adrenal Hyperplasia - Pipeline Insight, 2021

  • ID: 5237642
  • Drug Pipelines
  • January 2021
  • Region: Global
  • 60 pages
  • DelveInsight

FEATURED COMPANIES

  • BridgeBio
  • Crinetics Pharmaceuticals
  • Neurocrine BIosciences
  • Spruce Biosciences
This “Congenital Adrenal Hyperplasia - Pipeline Insight, 2021,” report provides comprehensive insights about 10+ companies and 10+ 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) refers to a group of disorders that arise from defective steroidogenesis. The production of cortisol in the zona fasciculata of the adrenal cortex occurs in five major enzyme-mediated steps. CAH results from deficiency in any one of these enzymes. Impaired cortisol synthesis leads to chronic elevations of ACTH via the negative feedback system, causing overstimulation of the adrenal cortex and resulting in hyperplasia and oversecretion of the precursors to the enzymatic defect. In general, all forms of CAH are transmitted in autosomal recessive mode of inheritance as a monogenic disorder. The most common enzyme deficiency that accounts for more than 90% of all CAH cases is 21-hydroxylase deficiency.

Symptoms

Many individuals with Congenital Adrenal Hyperplasia present with abnormally enlarged adrenal glands (hyperplastic adrenomegaly) that produce excessive amounts of androgens (male steroid hormones) leading to abnormal sexual development in females affected with the disease. Females with severe or classic virilizing CAH due to 21-hydroxylase deficiency will most likely have ambiguous or atypical external genitalia (masculinization or virilization), although they are genetically female and will have normal internal reproductive organs. Males with this type of CAH will not have ambiguous genitalia. Both genders can experience other symptoms such as early onset of puberty, fast body growth, and premature completion of growth leading to short stature, if they are not diagnosed and treated in early life.

Diagnosis

Potential diagnosis of CAH must be suspected in infants born with ambiguous genitalia. The physician is obliged to make the diagnosis as quickly as possible to initiate therapy. The diagnosis and rational decision of sex assignment must rely on the determination of genetic sex, the hormonal determination of the specific deficient enzyme, and an assessment of the patient's potential for future sexual activity and fertility. Diagnosis of the 21OHD CAH can also be confirmed biochemically by a hormonal evaluation. In a randomly timed blood sample, a very high concentration of 17-hydroxyprogesterone (17-OHP), the precursor of the defective enzyme, is diagnostic of classical 21OHD. The gold standard for hormonal diagnosis is the corticotropin stimulation test (250 μg cosyntropin intravenously), measuring levels of 17-OHP and Δ4-androstenedione at baseline and 60 min.

Treatment

Treatment of classical CAH starts soon after birth and is needed throughout the patient's life. People with classical CAH should have a team of healthcare providers, including specialists in pediatric endocrinology, uro-gynecologic surgery (for girls), psychology and genetics. People with classical CAH can have normal, fulfilling lives. The primary goal of treating classical CAH is to reduce the excess androgen production and replace the deficient hormones. Proper treatment with the correct dosage of these hormones is crucial to preventing adrenal crisis and virilization. Daily tablets including glucocorticoids (to replace cortisol), mineralocorticoids (to replace aldosterone) and salt supplements may be prescribed, particularly in infancy. Surgery may be considered to correct the appearance of ambiguous genitalia and/or relieve urethra-vaginal outlet obstruction that may lead to infection. Usually surgery is thought to be easier when performed at about 6-12 months after birth. Non-classical CAH on the other hand, is not life-threatening and relatively mild. People who have no obvious symptoms of non-classical CAH do not require surgery or medical treatment.

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 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.

Congenital Adrenal Hyperplasia Emerging Drugs

Crinecerfont: Neurocrine Biosciences

Crinecerfont is a corticotropin-releasing factor type 1 (CRF1) receptor antagonist under evaluation for the treatment of classic Congenital Adrenal Hyperplasia. It is being developed by Neurocrine Biosciences and is currently in Phase 3 of clinical trial. The study got initiated in July 2021 and is expected to get completed by February 2024 with 165 expected enrolled participants.

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. 10+ 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.

Phases

This report covers around 10+ 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
  • Oral
  • Parenteral
  • intravitreal
  • Subretinal
  • Topical.
  • Molecule Type
Products have been categorized under various Molecule types such as
  • Monoclonal Antibody
  • Peptides
  • Polymer
  • Small molecule
  • Gene therapy
  • Product Type
The 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.

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.
  • In December 2018, Millendo Therapeutics announced successful merger completion with OvaScience.
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 Answered

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
  • Spruce Biosciences
  • BridgeBio
  • Crinetics Pharmaceuticals
Key Products
  • Crinecerfont
  • Tildacerfont
  • BBP631
Research programme: ACTH antagonists

This report will be delivered within 1-3 business days
Note: Product cover images may vary from those shown

FEATURED COMPANIES

  • BridgeBio
  • Crinetics Pharmaceuticals
  • Neurocrine BIosciences
  • Spruce Biosciences
Introduction

Executive Summary

Congenital Adrenal Hyperplasia: Overview
  • Causes
  • Mechanism of Action
  • Signs and Symptoms
  • Diagnosis
  • Disease Management
Pipeline Therapeutics
  • Comparative Analysis
Therapeutic Assessment
  • Assessment by Product Type
  • Assessment by Stage and Product Type
  • Assessment by Route of Administration
  • Assessment by Stage and Route of Administration
  • Assessment by Molecule Type
  • Assessment by Stage and Molecule Type
Congenital Adrenal Hyperplasia - Analytical Perspective

In-depth Commercial Assessment
  • Congenital Adrenal Hyperplasia companies’ collaborations, Licensing, Acquisition - Deal Value Trends
Congenital Adrenal Hyperplasia Collaboration Deals
  • Company-Company Collaborations (Licensing / Partnering) Analysis
  • Company-University Collaborations (Licensing / Partnering) Analysis
Late Stage Products (Phase III)
  • Comparative Analysis
Crinecerfont: Neurocrine Biosciences
  • Product Description
  • Research and Development
  • Product Development Activities
Mid Stage Products (Phase II)
  • Comparative Analysis
Tildacerfont: Spruce Biosciences
  • Product Description
  • Research and Development
  • Product Development Activities
Early Stage Products (Phase I)
  • Comparative Analysis
Drug Name: Company Name
  • Product Description
  • Research and Development
  • Product Development Activities
Pre-clinical and Discovery Stage Products
  • Comparative Analysis
BBP631: BridgeBio
  • Product Description
  • Research and Development
  • Product Development Activities
Inactive Products
  • Comparative Analysis
Congenital Adrenal Hyperplasia Key Companies

Congenital Adrenal Hyperplasia Key Products

Congenital Adrenal Hyperplasia- Unmet Needs

Congenital Adrenal Hyperplasia- Market Drivers and Barriers

Congenital Adrenal Hyperplasia- Future Perspectives and Conclusion

Congenital Adrenal Hyperplasia Analyst Views

Congenital Adrenal Hyperplasia Key Companies

Appendix

List of Tables
Table 1 Total Products for Congenital Adrenal Hyperplasia
Table 2 Late Stage Products
Table 3 Mid Stage Products
Table 4 Early Stage Products
Table 5 Pre-clinical & Discovery Stage Products
Table 6 Assessment by Product Type
Table 7 Assessment by Stage and Product Type
Table 8 Assessment by Route of Administration
Table 9 Assessment by Stage and Route of Administration
Table 10 Assessment by Molecule Type
Table 11 Assessment by Stage and Molecule Type
Table 12 Inactive Products

List of Figures
Figure 1 Total Products for Congenital Adrenal Hyperplasia
Figure 2 Late Stage Products
Figure 3 Mid Stage Products
Figure 4 Early Stage Products
Figure 5 Preclinical and Discovery Stage Products
Figure 6 Assessment by Product Type
Figure 7 Assessment by Stage and Product Type
Figure 8 Assessment by Route of Administration
Figure 9 Assessment by Stage and Route of Administration
Figure 10 Assessment by Molecule Type
Figure 11 Assessment by Stage and Molecule Type
Figure 12 Inactive Products
Note: Product cover images may vary from those shown
  • Neurocrine BIosciences
  • Spruce Biosciences
  • BridgeBio
  • Crinetics Pharmaceuticals
Note: Product cover images may vary from those shown
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