#United States #San Francisco #Neurocrine Biosciences #Congenital Adrenal Hyperplasia #CAHtalog

Neurocrine Biosciences Unveils CAHtalog® Study Results

At ENDO 2025, Neurocrine Biosciences, Inc. has shared new insights from the CAHtalog® registry which reveal pressing challenges in the treatment of patients with classic congenital adrenal hyperplasia (CAH). The study highlighted alarming statistics: nearly 95% of adults and children involved in the registry experienced high levels of glucocorticoid exposure along with longstanding health complications associated with suboptimal disease control.

A Closer Look at the Findings

The findings presented at the Endocrine Society’s Annual Meeting in San Francisco underline significant variability in glucocorticoid (GC) treatment regimens. There were constant changes observed in health outcomes over time for both pediatric and adult patients. According to Dr. Sanjay Keswani, Chief Medical Officer of Neurocrine Biosciences, the data illustrated the chaotic and unpredictable nature of disease control for individuals suffering from classic CAH. Most participants experienced elevated glucocorticoid exposure or loss of androgen control multiple times throughout their lives.

In the analysis of 98 participants (totaling 37 adults and 42 children and adolescents, with 19 displaying data as both), researchers measured glucocorticoid treatments along with levels of androstenedione (A4). The patterns derived from this detailed data analysis emphasized how recurrent issues in hormone regulation and treatment intensity can complicate disease management in the long run, even with continuous glucocorticoid therapy.

The reality of managing CAH highlighted by this study is stark: achieving a temporary health state with low GC doses can be fleeting. Among those eligible for transition analysis, a striking 88.9% underwent at least one health state change, and over half underwent three or more transitions during their observation period, which averaged 8.6 years. Almost all participants (95.2%) fell into a suboptimal health state at some point, revealing high doses of GC use or elevated A4 levels.

The Need for Improved Therapies

As elucidated by Dina Matos, Executive Director of the CARES Foundation, managing CAH is a constantly evolving challenge. Even when glucocorticoids serve to momentarily control symptoms, sustaining this control remains a prominent issue. Such complexities necessitate new, innovative treatments to ease the burden on CAH patients.

The analysis also noted the need for alternative treatments, as high-dose glucocorticoid therapies are often associated with serious side effects, including metabolic disturbances and psychological impacts that can significantly affect a patient’s quality of life long term. Furthermore, adverse outcomes such as cardiovascular problems and abnormal growth in pediatric patients are also concerning implications of the existing treatment paradigms.

Embracing Advanced Therapeutic Options

The conversation around the presentation at ENDO 2025 also centered on the potential benefits of new treatments in the pipeline. One such emerging therapy, CRENESSITY® (crinecerfont), was discussed in relation to reducing both ACTH and adrenal androgens without necessitating high glucocorticoid doses. This medication could revolutionize CAH treatment, providing more physiological options and potentially mitigating the long-term side effects associated with traditional glucocorticoid therapies.

Given the findings from the CAHtalog registry, it’s evident that there is an urgent call for continued monitoring and further advancements in treatment methodologies aimed at enhancing patient outcomes. Neuocrine Biosciences is at the forefront of this initiative, offering substantial hope for patients grappling with this challenging condition.

With robust data leading the way, the potential for creative, patient-centered therapies underscores Neurocrine’s mission to advance healthcare solutions to better address the intricate needs of patients living with CAH. As the scientific community rallies around these findings, the spotlight remains on the urgent need for transformation in the treatment landscape of congenital adrenal hyperplasia.