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Neurocrine Biosciences Receives FDA Approval for CRENESSITY™

Neurocrine Biosciences, Inc. has officially announced the FDA's approval of CRENESSITY™ (crinecerfont), a pioneering treatment aimed at individuals with Classic Congenital Adrenal Hyperplasia (CAH). This drug is recognized as the first major advancement in 70 years for this rare genetic disorder, which affects both adults and children.

What is CRENESSITY?

CRENESSITY is a potent oral medication designed to enhance the quality of life for those managing CAH. The medication acts primarily as a selective antagonist of the corticotropin-releasing factor type 1 receptor (CRF1), helping to control the levels of excess adrenal hormones in both pediatric patients, aged four and older, and adults. This innovative treatment serves as an adjunct therapy alongside traditional glucocorticoid replacement therapy.

Clinical Significance

The approval is underpinned by compelling results from the expansive CAHtalyst clinical trial program, which represented the largest ever study on CAH involving both pediatric and adult populations. Dr. Kyle W. Gano, the CEO of Neurocrine Biosciences, emphasized the importance of this milestone, recognizing the invaluable contributions of all participants in the clinical trials and the support from their families.

Neurocrine's relentless commitment to understanding the complexities of CAH has led them to the forefront of treatment innovation. Patients and families often face a struggle to manage not only the symptoms of CAH but also the side effects associated with high-dose cortisol treatments, which can significantly affect their quality of life.

Dina Matos, Executive Director of the CARES Foundation, noted, “We are grateful to Neurocrine Biosciences for engaging with our community throughout the drug development process to understand our needs.” The advent of CRENESSITY aims to alleviate the burden of glucocorticoid treatment, reducing both androgen levels and the need for high steroid dosages.

Availability and Access

CRENESSITY is set to be commercially available within a week following its approval, with distribution handled through PANTHERx Rare, a specialty pharmacy focused on streamlining prescription processes for rare conditions. Neurocrine Biosciences also announced a support program called Neurocrine Access Support, aiming to assist patients and caregivers in navigating insurance processes and securing financial aid. Most patients can expect to pay $10 or less monthly for CRENESSITY, making treatment more accessible for families facing CAH.

CAHtalyst Studies Findings

The CAHtalyst clinical studies illuminated the drug's benefits:

• - In the pediatric cohort, patients experienced a significant decrease in androstenedione levels compared to those receiving a placebo. Children taking the medication notably reduced their glucocorticoid doses while maintaining androgen control.
• - In adult participants, 63% achieved glucocorticoid doses within the physiological range while either maintaining or improving androgen levels.
• - The adult study showcased that patients on CRENESSITY had significantly lower levels of both androstenedione and 17-hydroxyprogesterone, critical hormones affected in CAH.

Despite the promising results, some patients reported mild to moderate side effects, including headache, fatigue, and abdominal pain, which were generally manageable. Importantly, the frequency of serious adverse events was low, demonstrating a favorable safety profile for CRENESSITY compared to traditional treatments.

Understanding Congenital Adrenal Hyperplasia

Congenital adrenal hyperplasia (CAH) results from genetic variations affecting adrenal hormone production. Most cases derive from the CYP21A2 gene mutations that result in 21-hydroxylase enzyme deficiency, impacting cortisol and aldosterone synthesis while simultaneously producing excess adrenal androgens. Untreated CAH poses significant health risks, including dehydration and critical electrolyte imbalances. Historically, glucocorticoid therapy was the standard treatment but often resulted in complications from overtreatment.

Neurocrine's CRENESSITY is a groundbreaking step toward more effective management of CAH, allowing for lower glucocorticoid doses and potentially mitigating associated long-term health risks. The industry's shift towards this advanced therapy could fundamentally change how patients experience their treatment journey.

For more information about CRENESSITY, visit Crenessity.com.