Neurocrine’s New CAH Drug, Krenicity, Gets FDA Approval—Here’s What You Need to Know

In a significant advancement for rare disease treatment, the U.S. Food and Drug Administration (FDA) has approved Neurocrine Biosciences’ new therapy for congenital adrenal hyperplasia (CAH).

The FDA recently approved Krenicity, a novel therapy by Neurocrine Biosciences designed specifically for typical CAH. This approval represents a pivotal milestone in the management of this rare condition.

CAH is a rare inherited disorder that affects the adrenal glands. These glands produce essential hormones such as cortisol and androgens. Patients with CAH often fail to produce sufficient cortisol, which is critical for stress regulation, while overproducing androgen hormones similar to testosterone.

To compensate for these hormonal imbalances, CAH patients typically rely on higher-than-normal doses of glucocorticoids to supplement cortisol deficiency and suppress excessive androgen production.

The FDA has approved Krenicity for adults and children aged 4 and older with CAH to be used alongside standard glucocorticoid therapy. A Neurocrine spokesperson stated that Krenicity will become commercially available within a week.

Krenicity’s mechanism of action involves reducing the overproduction of androgens, which in turn allows for lower glucocorticoid dosage requirements.

Neurocrine’s clinical trials showed promising results. In a 24-week study involving 122 adult CAH patients, those treated with Krenicity twice daily successfully reduced their daily glucocorticoid dose by up to 27% while maintaining stable levels of androstenedione, a key androgen hormone. These outcomes contrasted with those of the 60 patients who received a placebo.

The drug demonstrated similar efficacy in pediatric patients. In a 28-week trial involving 69 children with CAH, participants treated with Krenicity twice daily maintained stable hormone levels despite reducing their daily glucocorticoid dose by 18%, compared to the placebo group.