CYP11B1

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. Related Reports Congenital Adrenal Hyperplasia Pipeline Congenital Adrenal Hyperplasia Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key CAH companies, including Spruce Biosciences, Lundbeck, among others. Congenital Adrenal Hyperplasia Epidemiology Forecast Congenital Adrenal Hyperplasia Epidemiology Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, and CAH epidemiology trends. Adrenal Crisis Market Adrenal Crisis Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, market share of the individual therapies, and key adrenal crisis companies including Eton Pharmaceuticals, Antares Pharma , among others. Adrenal Crisis Pipeline Adrenal Crisis Pipeline Insight – 2024 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key adrenal crisis companies, including Eton Pharmaceuticals, Antares Pharma, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

ESTEVE has completed the acquisition of HRA Pharma Rare Diseases, a business specialized in rare diseases, from Perrigo Company plc. With this acquisition, ESTEVE expands its portfolio in rare and severe diseases adding, to the current existing offer, three new medications to treat Cushing's syndrome and adrenocortical carcinoma: Metopirone, Lysodren, and Ketoconazole HRA. HRA Pharma Rare Diseases has been a leading company for over 15 years in rare and severe diseases, providing the best care and services to individuals living with rare diseases, and is committed to supporting healthcare professionals worldwide. "This acquisition allows us to accelerate our international expansion, strengthening our global presence and ability to better address unmet needs, thus improving the lives of more people," highlights Staffan Schüberg, Chief Executive Officer of ESTEVE. With annual net revenues in 2023 of approximately 50 million euros, HRA Pharma Rare Diseases has a presence in numerous European countries and the United States with a team of 49 individuals. This agreement represents an investment of up to 275 million euros, structured with an initial fixed contribution and future variable payments tied to revenue. Share

ESTEVE has signed a binding offer with Perrigo Company plc to acquire Perrigo's HRA Pharma Rare Diseases, a business specialized in rare and ultra-rare diseases, following the information and consultation process with HRA Pharma Works Council in France and subject to the satisfaction of customary closing conditions, including receipt of regulatory approvals. ESTEVE will increase its portfolio in the rare and severe diseases therapeutic area, adding to its current portfolio three new medicines that address Cushing's syndrome and Adrenocortical Carcinoma: Metopirone, Lysodren, and Ketoconazole HRA. HRA Pharma Rare Diseases has over 15 years' experience in rare and ultra-rare diseases and is dedicated to bringing the best care and services to people living with rare diseases and committed to supporting healthcare professionals all over the world. With annual Net Revenues in 2023 around €50M, the HRA Pharma Rare Diseases business has a presence in several European countries as well as in the US. "This transaction aims to advance ESTEVE on the path of covering the unmet patients' needs, in line with ESTEVE's purpose of improving people's lives and is another step towards the company's vision of being an international specialty pharma company", concludes Staffan Schüberg, Chief Executive Officer of ESTEVE. Patrick Lockwood-Taylor, Perrigo's President and Chief Executive Officer, states: "We are pleased that ESTEVE, with its track record of growth and international footprint, will look to increase global access to these effective treatment options for all those patients in need". The binding offer is for up to €275 million, consisting of an upfront payment and potential earnout payments. The closing of the transaction should take place during the third quarter of 2024, following the required works council consultation and subject to customary regulatory approvals. Perella Weinberg Partners and Clifford Chance served as ESTEVE's lead advisors on this transaction. Morgan Stanley & Co. LLC and Wachtell, Lipton, Rosen & Katz served as Perrigo's lead advisors on this transaction.