Congenital Adrenal Hyperplasia Pipeline Analysis: 5+ Companies are Working to Improve the Treatment Space | DelveInsight

DelveInsight's, "Congenital Adrenal Hyperplasia Pipeline Insight, 2022," report provides comprehensive insights about 5+ companies and 5+ 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.

Key takeaways from the Congenital Adrenal Hyperplasia Pipeline
• DelveInsight's Congenital Adrenal Hyperplasia Pipeline report depicts a robust space with 5+ active players working to develop 5+ pipeline therapies for Congenital Adrenal Hyperplasia.
• The leading Congenital Adrenal Hyperplasia Companies are working such as Neurocrine Biosciences, Spruce Biosciences, Adrenas Therapeutics Inc, Crinetics Pharmaceuticals, Diurnal, and others.
• Promising Congenital Adrenal Hyperplasia Pipeline therapies such as Crinecerfont, ATR-101, AAV BBP-631, Chronocort, Cortef, SPR001, NBI-74788, and others.
• The Congenital Adrenal Hyperplasia 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.
Recent Developmental Activities in the Congenital Adrenal Hyperplasia Pipeline
• The US Food and Drug Administration (FDA) has agreed a Special Protocol Assessment (SPA) for Chronocort (modified-release hydrocortisone) for the design, endpoints and statistical analysis approach of a pivotal study of Chronocort for the treatment for Congenital Adrenal Hyperplasia (CAH) in the US. The SPA provides agreement that the Phase III trial design adequately addresses objectives that would support the regulatory submission for drug approval.

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Congenital Adrenal Hyperplasia Overview

Congenital adrenal hyperplasia (CAH) is a group of rare inherited autosomal recessive disorders characterized by a deficiency of one of the enzymes needed to make specific hormones. CAH affects the adrenal glands located at the top of each kidney. Normally, the adrenal glands are responsible for producing three different hormones: 1. corticosteroids, which gauge the body's response to illness or injury; 2. mineralocorticoids, which regulate salt and water levels; and 3. androgens, which are male sex hormones. An enzyme deficiency will make the body unable to produce one or more of these hormones, which in turn will result in the overproduction of another type of hormone precursor in order to compensate for the loss. The most common cause of CAH is the absence of the enzyme 21-hydroxylase. Different mutations in the gene responsible for 21-hydroxylase result in different levels of the enzyme, producing a spectrum of effects. Genetic testing for the gene mutations associated with the various forms of CAH is available, but is most often performed when pre-pregnancy genetic counseling is indicated, after an endocrinologist confirms the diagnosis through blood hormone tests, or if results of hormone tests are not definitive. Treatment of CAH varies greatly depending on the type and severity. CAH cannot be cured, but it can be effectively treated. The primary goal of treating classical CAH is to reduce the excess androgen production and replace the deficient hormones.

Congenital Adrenal Hyperplasia Emerging Drugs Profile
• Chronocort: Diurnal Group PLC
Congenital Adrenal Hyperplasia Pipeline Therapeutic Assessment
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. Registered include, Diurnal Group plc.

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Scope of the Congenital Adrenal Hyperplasia Pipeline Report
• Coverage- Global
• Congenital Adrenal Hyperplasia Pipeline Therapeutic Assessment By Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III
• Congenital Adrenal Hyperplasia Companies- Neurocrine Biosciences, Spruce Biosciences, Adrenas Therapeutics Inc, Crinetics Pharmaceuticals, Diurnal, and others.
• Congenital Adrenal Hyperplasia Pipeline therapies- Crinecerfont, ATR-101, AAV BBP-631, Chronocort, Cortef, SPR001, NBI-74788, and others.

Table of Content
1. Introduction
2. Congenital Adrenal Hyperplasia Executive Summary
3. Congenital Adrenal Hyperplasia: Overview
4. Congenital Adrenal Hyperplasia Pipeline Therapeutics
5. Congenital Adrenal Hyperplasia Therapeutic Assessment
6. Late Stage Products (Registered)
7. Chronocort: Diurnal Group plc
8. Drug profiles in the detailed report…..
9. Mid Stage Products (Phase II)
10. SPR001: spruce Biosciences
11. Drug profiles in the detailed report…..
12. Early stage products (Phase I/II)
13. AAV BBP-631: Adrenas Therapeutics Inc
14. Drug profiles in the detailed report…..
15. Inactive Products
16. Congenital Adrenal Hyperplasia Key Companies
17. Congenital Adrenal Hyperplasia Key Products
18. Congenital Adrenal Hyperplasia- Unmet Needs
19. Congenital Adrenal Hyperplasia- Market Drivers and Barriers
20. Congenital Adrenal Hyperplasia- Future Perspectives and Conclusion
21. Congenital Adrenal Hyperplasia Analyst Views
22. Congenital Adrenal Hyperplasia Key Companies
23. Appendix

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?

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