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.
LAS VEGAS, Sept. 30, 2024 /PRNewswire/ -- CAH refers to a rare set of inherited autosomal recessive disorders that impair hormone production in the adrenal glands, which sit on top of the kidneys. Normally, these glands produce three types of hormones: corticosteroids, mineralocorticoids, and androgens. CAH is mostly caused by mutations in the 21-hydroxylase gene, which accounts for more than 90% of cases. In rare instances, mutations in the 11-hydroxylase gene can also lead to CAH.
In the 7MM, the US accounted for the highest prevalent cases of congenital adrenal hyperplasia in 2023, with around
50% of the total prevalent cases across the 7MM; these numbers are expected to increase during the forecast period (2024–2034).
DelveInsight's analysis reveals that a higher prevalence of diagnosed congenital adrenal hyperplasia is observed in the
18-year-old
and above age group across the 7MM. Among mutation-based congenital adrenal hyperplasia cases,
21 OHD (CYP21A2 gene mutation) represents a higher prevalence compared to
11-Beta hydroxylase (CYP11B1 gene mutation), and others.
The approach to managing CAH differs significantly based on its type and severity. While CAH cannot be completely cured, it can be managed effectively. For classical CAH, treatment begins shortly after birth and continues throughout the individual's life. Those with classical CAH should work with a team of healthcare professionals, including experts in pediatric endocrinology, uro-gynecologic surgery, psychology, and genetics. With appropriate care, individuals with classical CAH can lead normal and satisfying lives. In contrast, treatment for non-classical CAH may not be necessary, depending on the severity of symptoms.
The management of classic CAH focuses on three main objectives: correcting adrenal insufficiency, lowering adrenal androgens to appropriate levels, and preventing iatrogenic hypercortisolism. Hydrocortisone is the primary glucocorticoid used, administered two to three times daily with higher doses in the morning and lower doses in the evening to align with the body's natural circadian rhythm. For NC-CAH, cortisol replacement is generally unnecessary, except in specific situations such as adult women with hyperandrogenism and fertility problems, or pediatric patients experiencing rapidly advancing precocious puberty. Currently, the only medication approved in Europe for CAH treatment is
EFMODY (Hydrocortisone Modified-release Hard Capsules) by
Neurocrine Biosciences.
Learn more about the FDA-approved congenital adrenal hyperplasia drugs @
Drugs for Congenital Adrenal Hyperplasia Treatment
EFMODY replicates the natural circadian rhythm of cortisol secretion. It compensates for low cortisol levels in patients with congenital adrenal hyperplasia, aiding in the management of the condition's symptoms. Approved for use in adolescents aged 12 and older, as well as adults, EFMODY uses hydrocortisone—a glucocorticoid that affects various tissues through intracellular steroid receptors.
Containing synthetic cortisol, EFMODY gradually releases it in the intestines to mimic the body's natural cortisol release, helping to restore hormone balance and alleviate condition-related issues.
In May 2021, the EMA approved EFMODY for treating congenital adrenal hyperplasia in patients aged 12 and above. Diurnal launched the drug in Germany and Austria in September 2021, and later in the United Kingdom. In November 2022, Neurocrine Biosciences acquired Diurnal for approximately
USD 55 million.
To know more about congenital adrenal hyperplasia treatment options, visit @
New Treatment for Congenital Adrenal Hyperplasia
The congenital adrenal hyperplasia pipeline is strong with many companies working in the domain. Various potential therapies that are projected to enter during the forecast period include
Neurocrine Biosciences' Crinecerfont, Spruce Biosciences' Tildacerfont (SPR001), Crinetics Pharmaceuticals' Atumelnant (CRN04894), BridgeBio's BBP-631, Lundbeck's Lu AG13909, and others.
Discover which therapies are expected to grab major congenital adrenal hyperplasia market share @
Congenital Adrenal Hyperplasia Market Report
Crinecerfont is an investigational oral medication that acts as a selective antagonist for the corticotropin-releasing factor Type 1 receptor (CRF1). It's being developed to manage and regulate high levels of Adrenocorticotropic Hormone (ACTH) and adrenal androgens without relying on glucocorticoids, specifically for treating congenital adrenal hyperplasia caused by 21-hydroxylase deficiency. By blocking the CRF1 receptor, Crinecerfont reduces the overproduction of ACTH, which in turn decreases the excess production of androgens seen in CAH patients. This mechanism aims to normalize hormone levels and address the root cause of the condition.
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.
The NDAs for crinecerfont include (1) a capsule formulation (NDA# 218808) and (2) an oral solution formulation (NDA# 218820), both addressing the efficacy and safety of crinecerfont for classic CAH. The FDA has set Prescription Drug User Fee Act (PDUFA) target action dates for these applications as
December 29 and December 30, 2024, respectively. The FDA has indicated it does not plan to hold an advisory committee meeting regarding these NDAs.
Crinecerfont received an
Orphan Drug designation
(ODD) in March 2019 and a
Breakthrough Therapy designation (BTD) in December 2023. The results from the
CAHtalyst Phase III trial, involving both pediatric and adult patients with CAH caused by 21-OHD, were published online in The New England Journal of Medicine on June 2 and June 1, respectively, and were also presented at ENDO 2024.
BBP-631 is a gene therapy in development using adeno-associated virus (AAV) to target the root cause of congenital adrenal hyperplasia due to 21-hydroxylase deficiency. This therapy aims to deliver a functional copy of the 21-hydroxylase gene directly to the adrenal glands, where hormones are produced. Animal studies have demonstrated its effectiveness in providing consistent and long-term delivery to the adrenal glands. If successful, BBP-631 could help restore natural hormone and steroid levels in people with CAH, allowing them to produce cortisol and aldosterone—something no current treatments or clinical trials can achieve. BridgeBio envisions this gene therapy as a potential solution to correct the hormonal imbalance caused by CAH.
Tildacerfont is a new-generation antagonist targeting the Corticotropin-releasing factor (CRF1) receptor. It aims to significantly enhance disease management and decrease reliance on steroids for patients with classic congenital adrenal hyperplasia and other conditions associated with high ACTH levels. By binding to CRF1 receptors, tildacerfont reduces the overproduction of ACTH and, consequently, lowers the production of adrenal androgens like androstenedione in patients with CAH and Polycystic Ovary Syndrome (PCOS).
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.
Discover more about drugs for congenital adrenal hyperplasia in development @
Congenital Adrenal Hyperplasia Clinical Trials
The anticipated launch of these emerging therapies for congenital adrenal hyperplasia are poised to transform the market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the congenital adrenal hyperplasia market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth.
DelveInsight estimates that the market size for congenital adrenal hyperplasia is expected to grow from
USD 20 billion in 2023 with a tremendous
CAGR of ~40% by 2034. This growth can be attributed to the introduction of upcoming therapies and the rising prevalence of the disease.
DelveInsight's latest published market report titled as
Congenital Adrenal Hyperplasia Market Insight, Epidemiology, and Market Forecast – 2034 will help you to discover which market leader is going to capture the largest market share. The report provides comprehensive insights into the congenital adrenal hyperplasia country-specific treatment guidelines, patient pool analysis, and epidemiology forecast to help understand the key opportunities and assess the market's underlying potential. The congenital adrenal hyperplasia market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into:
Total Diagnosed Prevalent Cases of Congenital Adrenal Hyperplasia
Gender-specific Cases of Congenital Adrenal Hyperplasia
Age-specific Cases of Congenital Adrenal Hyperplasia
Mutation-based Cases of Congenital Adrenal Hyperplasia
Type-specific Cases of Congenital Adrenal Hyperplasia
The report provides an edge while developing business strategies by understanding trends shaping and driving the 7MM congenital adrenal hyperplasia market. Highlights include:
11-year Forecast
7MM Analysis
Epidemiology-based Market Forecasting
Historical and Forecasted Market Analysis up to 2034
Emerging Drug Market Uptake
Peak Sales Analysis
Key Cross Competition Analysis
Industry Expert's Opinion
Access and Reimbursement
Download this congenital adrenal hyperplasia market report to assess the epidemiology forecasts, understand the patient journeys, know KOLs' opinions about the upcoming treatment paradigms, and determine the factors contributing to the shift in the congenital adrenal hyperplasia market. Also, stay abreast of the mitigating factors to improve your market position in the congenital adrenal hyperplasia therapeutic space.
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