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Congenital Adrenal Hyperplasia Drugs Market

(Albany, USA) DelveInsight’s Congenital Adrenal Hyperplasia Market Insights report includes a comprehensive understanding of current treatment practices, congenital adrenal hyperplasia emerging drugs, market share of individual therapies, and current and forecasted market size from 2020 to 2034, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan].

The congenital adrenal hyperplasia market report covers emerging drugs, treatment practices, market share of the individual therapies, and current & forecasted market size from 2020 to 2034. It also evaluates the current treatment practice/algorithm, market drivers & barriers, and unmet medical needs to curate the best of opportunities and assess the underlying potential of the congenital adrenal hyperplasia market.

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Key Takeaways from the Congenital Adrenal Hyperplasia Market Report

• As per DelveInsight analysis, total Congenital Adrenal Hyperplasia Market Size in 2023 was more than USD 20 million in the 7MM, which is expected to grow at a CAGR of ~40% during the forecast period (2020-2034).
• As per DelveInsight estimates, the total 7MM diagnosed prevalent cases of CAH in 2021 were more than 73K out of which the maximum cases were seen in the US.
• Leading congenital adrenal hyperplasia companies such as Crinetics Pharmaceuticals Inc., Diurnal Limited, Adrenas Therapeutics Inc, Neurocrine Biosciences, Lundbeck, Bridgebio, Spruce Biosciences, and others are developing novel congenital adrenal hyperplasia drugs that can be available in the congenital adrenal hyperplasia market in the coming years.
• The promising congenital adrenal hyperplasia therapies in the pipeline include CRN04894, Chronocort, EFMODY, AAV BBP-631, Crinecerfont, Tildacerfont, ATUMELNANT, BBP-631, Lu AG13909 and others.
• In May 2025, Neurocrine Biosciences, Inc. (Nasdaq: NBIX) has released new findings from its Phase 3 CAHtalyst™ Adult and Pediatric trials evaluating CRENESSITY™ (crinecerfont). The results revealed that a significant number of pediatric patients with classic congenital adrenal hyperplasia (CAH) reached glucocorticoid doses within the physiological range and achieved normal androstenedione levels. Furthermore, adult male patients showed improvements in certain reproductive hormone levels. Both groups—adults and children—experienced notable reductions in glucocorticoid dosing. These findings were shared at the 2025 Annual Meeting of the American Association of Clinical Endocrinology in Orlando.
• In January 2025, Crinetics Pharmaceuticals, Inc. (Nasdaq: CRNX) today announced positive topline results from an open-label, Phase 2 congenital adrenal hyperplasia (CAH) study of investigational atumelnant, a novel, once-daily oral adrenocorticotropic hormone (ACTH) receptor antagonist candidate being developed for the treatment of classic CAH and ACTH-dependent Cushing’s syndrome.
• In September 2024, BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a commercial-stage biopharmaceutical company focused on genetic diseases, today announced topline results from the Phase 1/2 open-label ADventure study investigating BBP-631, the Company’s investigational adeno-associated virus (AAV) 5 gene therapy, for the treatment of congenital adrenal hyperplasia (CAH).
• In August 2024, Spruce Biosciences announced that the top-line results from the CAHmelia-204 study, which evaluates tildacerfont in adult CAH patients, and the CAHptain-205 study, which assesses the drug in both adult and pediatric CAH patients, are expected to be released in the fourth quarter of 2024.
• In July 2024, Neurocrine Biosciences announced that the FDA has accepted its two New Drug Applications (NDAs) for crinecerfont, both with Priority Review designations. These applications are for treating classic congenital adrenal hyperplasia in children, adolescents, and adults. If approved, crinecerfont would become the first new treatment for CAH in 70 years and a pioneering therapy with a novel approach for this rare endocrine disorder.

Discover which therapies are expected to grab the major congenital adrenal hyperplasia market share @ Congenital Adrenal Hyperplasia Market Report

Congenital Adrenal Hyperplasia Overview

Congenital Adrenal Hyperplasia refers to a group of inherited genetic disorders affecting the adrenal glands, which are responsible for producing essential hormones like cortisol, aldosterone, and androgens. The most common form, accounting for over 90% of cases, is due to a deficiency of the enzyme 21-hydroxylase. This enzyme defect impairs cortisol and aldosterone synthesis, leading to excessive production of androgens (male hormones), which can result in varying degrees of virilization.

Congenital Adrenal Hyperplasia is typically classified into two types: classic and non-classic. Classic Congenital Adrenal Hyperplasia, often diagnosed in infancy or early childhood, includes salt-wasting and simple virilizing forms. Salt-wasting Congenital Adrenal Hyperplasia can cause life-threatening electrolyte imbalances and requires immediate treatment. Non-classic Congenital Adrenal Hyperplasia is a milder, late-onset form that may present in adolescence or adulthood with symptoms such as hirsutism, irregular menstruation, or infertility.

Symptoms depend on the severity and form of Congenital Adrenal Hyperplasia and may include ambiguous genitalia in newborn females, rapid growth in childhood, early puberty, and infertility. Diagnosis is confirmed through hormonal blood tests and genetic testing. Treatment typically involves lifelong hormone replacement therapy to manage hormone imbalances and prevent complications. With appropriate care, individuals with Congenital Adrenal Hyperplasia can lead healthy, productive lives.

Congenital Adrenal Hyperplasia Epidemiology Segmentation

United States has the highest number of age-specific CAH cases (more than 9K cases in

The congenital adrenal hyperplasia market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into:

• Congenital Adrenal Hyperplasia Diagnosed Prevalence
• Congenital Adrenal Hyperplasia Age-specific Cases
• Congenital Adrenal Hyperplasia Mutations-based Cases
• Type-specific Cases of Classical Congenital Adrenal Hyperplasia
• Congenital Adrenal Hyperplasia Treatment Market

While there is currently no cure for CAH, available treatments aim to alleviate associated congenital adrenal hyperplasia symptoms. Hormone replacement medications play a crucial role, elevating deficient hormone levels and moderating excess male hormone production. Individuals with classic CAH often require lifelong hormone medication, whereas those with milder forms may, in some cases, discontinue treatment in adulthood and maintain lower-dose regimens. Some individuals with non-classic CAH may not necessitate any form of congenital adrenal hyperplasia treatment.

Glucocorticoid replacement therapy aims to replace deficient steroids, control excess adrenal hormones and glucocorticoids, prevent virilization, optimize growth, and support fertility. The treatment of choice for growing children involves oral hydrocortisone tablets, while the use of hydrocortisone suspension is advised against. In cases of CAH, the primary approach to glucocorticoid replacement therapy typically utilizes oral hydrocortisone (10–15 mg/m2/24 h), administered in two or three divided doses per day.

For individuals diagnosed with the salt-wasting form of 21-OHD CAH, it is essential to undergo treatment involving 9α-fludrohydrocortisone (Florinef) at a daily dosage of 0.05–0.2 mg orally, along with sodium chloride supplementation at a rate of 1–2 g/day added to formula or foods. It is advisable for all individuals with the classic form of CAH to receive combined treatment with both 9α-fludrohydrocortisone and sodium chloride supplements during the newborn and early infancy stages.

To know more about congenital adrenal hyperplasia treatment guidelines, visit @ Congenital Adrenal Hyperplasia Management

Congenital Adrenal Hyperplasia Pipeline Therapies and Key Companies

• CRN04894: Crinetics Pharmaceuticals Inc.
• Chronocort: Diurnal Limited
• AAV BBP-631: Adrenas Therapeutics Inc
• Crinecerfont: Neurocrine Biosciences
• Tildacerfont: Spruce Biosciences

Congenital Adrenal Hyperplasia Market Dynamics

The dynamics of the congenital adrenal hyperplasia are expected to change in the coming years. In developed countries worldwide, prenatal and newborn screening programs for CAH disease have been successfully implemented, facilitating early detection and treatment in children. Ongoing research and therapeutic trials exploring novel molecules like selective corticotropin-releasing factor type 1-receptor inhibitors, gene therapy, and ACAT-1 inhibitors are yielding promising outcomes. A novel extended therapy approach is being explored to address the long-term side effects, complications, and circadian rhythm insufficiency associated with glucocorticoids and mineralocorticoids. Although the current treatment primarily targets patients with 21-hydroxylase insufficiency, potential research on atypical CAH variations is expected to open avenues for treating rare types in the future.

Furthermore, the congenital adrenal hyperplasia pipeline is very robust; many potential therapies are being investigated for the treatment of congenital adrenal hyperplasia, and it is safe to predict that the treatment space will significantly impact the congenital adrenal hyperplasia market during the forecast period. Moreover, the anticipated introduction of emerging therapies with improved efficacy and a further improvement in the diagnosis rate are expected to drive the growth of the congenital adrenal hyperplasia market in the 7MM.

However several factors may impede the growth of the congenital adrenal hyperplasia market. The diagnosis of non-classical CAH may not occur until a person begins to show symptoms in later stages of life. CAH can be misdiagnosed with other diseases such as Addison’s disease, Ovotesticular disorder, and Turner syndrome. Furthermore, gene therapy, while promising, is costly; moreover, the health system in most countries, including the US, is not set up for large one-time payments. In comparison to small molecules, gene therapies take a longer time for their approval, and the approval guidelines for gene therapies are not as well defined as those for small molecules or monoclonal antibodies.

Moreover, congenital adrenal hyperplasia treatment poses a significant economic burden and disrupts patients’ overall well-being and QOL. Furthermore, the congenital adrenal hyperplasia market growth may be offset by failures and discontinuation of emerging therapies, unaffordable pricing, market access and reimbursement issues, and a shortage of healthcare specialists. In addition, the undiagnosed, unreported cases and the unawareness about the disease may also impact the congenital adrenal hyperplasia market growth.

Learn more about the FDA-approved drugs for congenital adrenal hyperplasia @ Drugs for Congenital Adrenal Hyperplasia Treatment

Congenital Adrenal Hyperplasia Market Report Metrics

• Study Period: 2020–2034
• Coverage: 7MM [the United States, the EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan].
• Congenital Adrenal Hyperplasia Market Size in 2023: USD 20 Million
• Key Congenital Adrenal Hyperplasia Companies: Crinetics Pharmaceuticals Inc., Diurnal Limited, Adrenas Therapeutics Inc, Neurocrine Biosciences, Lundbeck, Bridgebio, Spruce Biosciences, and others
• Key Pipeline Congenital Adrenal Hyperplasia Therapies: CRN04894, Chronocort, AAV BBP-631, Crinecerfont, Tildacerfont, and others.
• Therapeutic Assessment: Congenital Adrenal Hyperplasia current marketed and emerging therapies
• Congenital Adrenal Hyperplasia Market Dynamics: Key Market Forecast Assumptions of Emerging Congenital Adrenal Hyperplasia Drugs and Market Outlook
• Competitive Intelligence Analysis: SWOT analysis and Market entry strategies
• Unmet Needs, KOL’s views, Analyst’s views, Congenital Adrenal Hyperplasia Market Access and Reimbursement

Discover more about congenital adrenal hyperplasia drugs in development @ Congenital Adrenal Hyperplasia Clinical Trials

Table of Contents

1. Congenital Adrenal Hyperplasia Key Insights
2. Congenital Adrenal Hyperplasia Report Introduction
3. Congenital Adrenal Hyperplasia Overview at a Glance
4. Congenital Adrenal Hyperplasia Executive Summary
5. Congenital Adrenal Hyperplasia Key Events
6. Epidemiology and Market Forecast Methodology
7. Disease Background and Overview
8. Congenital Adrenal Hyperplasia Treatment and Management
9. Congenital Adrenal Hyperplasia Guidelines
10. Congenital Adrenal Hyperplasia Epidemiology and Patient Population
11. Patient Journey
12. Key Endpoints in Congenital Adrenal Hyperplasia
13. Congenital Adrenal Hyperplasia Marketed Drugs
14. Congenital Adrenal Hyperplasia Emerging Drugs
15. 7MM Congenital Adrenal Hyperplasia Market Analysis
16. Market Access and Reimbursement
17. KOL Views
18. Unmet Needs
19. SWOT Analysis
20. Appendix
21. DelveInsight Capabilities
22. Disclaimer
23. About DelveInsight

About Us

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