(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].
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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.
- Neurocrine Biosciences has announced that the US Food and Drug Administration (FDA) has accepted its two New Drug Applications (NDA) for crinecerfont, granting them Priority Review designations. The applications cover the treatment of children, adolescents, and adults with classic congenital adrenal hyperplasia (CAH). If approved, crinecerfont would represent the first new treatment for CAH in 70 years and the first-in-class therapy offering a novel mechanism of action to address this rare and serious endocrine disorder.
- 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 (CAH) constitutes a rare cluster of autosomal recessive disorders stemming from an inherited deficiency in enzymes crucial for the production of specific hormones. The adrenal glands, nestled atop each kidney, play a pivotal role in synthesizing corticosteroids, mineralocorticoids, and androgens, with the absence of the enzyme 21-hydroxylase emerging as the primary cause of CAH. Certain forms of CAH warrant prenatal diagnosis methods like amniocentesis and CVS. Genetic testing takes precedence in scenarios involving pre-pregnancy genetic counseling, confirmation of diagnosis through blood hormone tests by an endocrinologist, or when hormone test results lack precision. Conversely, examinations encompassing genital, blood, and urine tests are employed to assess the production of hormones by the adrenal glands.
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 sex 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
- Congenital Adrenal Hyperplasia Key Insights
- Congenital Adrenal Hyperplasia Report Introduction
- Congenital Adrenal Hyperplasia Overview at a Glance
- Congenital Adrenal Hyperplasia Executive Summary
- Congenital Adrenal Hyperplasia Key Events
- Epidemiology and Market Forecast Methodology
- Disease Background and Overview
- Congenital Adrenal Hyperplasia Treatment and Management
- Congenital Adrenal Hyperplasia Guidelines
- Congenital Adrenal Hyperplasia Epidemiology and Patient Population
- Patient Journey
- Key Endpoints in Congenital Adrenal Hyperplasia
- Congenital Adrenal Hyperplasia Marketed Drugs
- Congenital Adrenal Hyperplasia Emerging Drugs
- 7MM Congenital Adrenal Hyperplasia Market Analysis
- Market Access and Reimbursement
- KOL Views
- Unmet Needs
- SWOT Analysis
- Appendix
- DelveInsight Capabilities
- Disclaimer
- About DelveInsight
About DelveInsight
DelveInsight is a leading Life Science market research and business consulting company recognized for its off-the-shelf syndicated market research reports and customized solutions to firms in the healthcare sector.
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