Neurocrine Biosciences, Inc. has announced the release of
CRENESSITY™ (crinecerfont) in the U.S. market, following its approval by the FDA as an additional treatment for
classic congenital adrenal hyperplasia (CAH). This first-of-its-kind oral medication is designed to be used alongside glucocorticoid therapy to manage androgen levels in patients aged four and above. CRENESSITY’s unique mechanism allows patients to reduce their glucocorticoid dosages while maintaining effective control over androgens, offering a new approach to
CAH management after decades of reliance on high-dose steroids.
The innovative treatment directly targets the
corticotropin-releasing factor type 1 receptor (CRF1) to decrease the production of adrenocorticotropic hormone (ACTH) and adrenal androgens. By doing so, it addresses the root causes of the condition without the complications linked to high-dose glucocorticoid therapy. This is particularly important given the long-term risks associated with
steroid excess, which include metabolic and cardiovascular issues, as well as potential psychological effects.
CRENESSITY is now available through a specialized distribution network, PANTHERx Rare, ensuring that patients have access to pharmacists who are specifically trained in CAH. These experts are available around the clock to assist patients, caregivers, and healthcare providers. Additionally, the Neurocrine Access Support program has been launched to provide comprehensive assistance to help patients navigate insurance and financial concerns, aiming to make the treatment accessible to a majority of patients at a manageable cost.
The approval of CRENESSITY came after extensive clinical trials—the largest ever conducted for classic CAH. The Phase 3 CAHtalyst™ studies included both pediatric and adult patients and demonstrated the treatment’s efficacy and safety. The pediatric study focused on patients aged four to 17, showing that CRENESSITY could improve androgen control, and allow for glucocorticoid dose reduction over 24 weeks. The adult study, involving participants aged 18 to 58, confirmed that CRENESSITY facilitated a similar reduction in glucocorticoid use, maintaining stable or improved androstenedione levels.
Congenital adrenal hyperplasia is a rare genetic disorder that disrupts steroid hormone production due to enzyme deficiencies, primarily affecting cortisol and aldosterone production. This condition often results in excessive androgen production, leading to significant health challenges if not managed properly. Traditional treatment methods relied on using higher-than-necessary doses of glucocorticoids to suppress androgen levels, which often led to severe side effects.
CRENESSITY offers a promising alternative by allowing for more physiologically appropriate dosing of glucocorticoids while effectively managing androgen levels. Available as both capsules and oral solutions, the treatment provides flexible dosing options based on age, weight, and individual patient needs. Patients are advised to continue their glucocorticoid therapy for cortisol replacement alongside CRENESSITY use.
Neurocrine Biosciences, the company behind CRENESSITY, has been at the forefront of developing treatments for under-addressed neuroendocrine and neurological disorders. Their commitment to advancing therapeutic options is evident in their diverse portfolio, which includes treatments for various complex conditions. With the introduction of CRENESSITY, Neurocrine Biosciences continues to expand its impact on patient care by offering innovative solutions to previously challenging medical conditions.
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