Neurocrine reports initial success with CRENESSITY in rare CAH subtype

The retrospective case series tracked 15 patients—11 pediatric and 4 adult—who suffer from 11β‑hydroxylase deficiency. This condition, which accounts for roughly 5% of classic CAH cases, is marked by the accumulation of specific adrenal steroid precursors, 11-deoxycortisol and 11-deoxycorticosterone, often leading to hypertension and other complications. Following the initiation of CRENESSITY, researchers observed substantial hormonal stabilization. Median levels of 11-deoxycorticosterone dropped by 92%, while 11-deoxycortisol decreased by 95%.

Beyond hormonal markers, the clinical impact was evident in daily management. Nearly all participants—14 out of 15—were able to lower their total glucocorticoid dosage. Furthermore, among the five patients previously managing hypertension with medication, two were able to reduce or entirely discontinue their antihypertensive therapy. These results provide preliminary evidence that CRENESSITY, by targeting ACTH through a non-glucocorticoid mechanism, may offer a viable therapeutic path for this specific patient population, potentially reducing the long-term risks associated with supraphysiologic steroid use.