On May 1 2026, Neurocrine Biosciences presented two‑year data from the Phase 3 CAHtalyst Pediatric study at the Pediatric Endocrine Society Annual Meeting in San Francisco, highlighting durable reductions in androgen levels and sustained decreases in glucocorticoid doses among children and adolescents with classic congenital adrenal hyperplasia (CAH).
The open‑label extension enrolled 86 participants aged four to 17 who completed up to 24 months of treatment. At 12 months, mean ACTH fell by 118 pg/mL and 17‑hydroxyprogesterone fell by 1,698 ng/dL, with further declines at 24 months. The mean daily glucocorticoid dose dropped from 16.4 mg/m²/day to 13.2 mg/m²/day, a 3.2 mg/m²/day reduction that reflects the drug’s ability to lower the need for steroid therapy.
Clinical benefits extended beyond hormone control. Body‑mass index standard deviation scores decreased by 0.24 at 12 months and 0.30 at 24 months, while insulin resistance measured by HOMA‑IR fell by 0.9 and 0.6, respectively. These improvements are linked to reduced steroid exposure, which is known to drive weight gain and insulin resistance in CAH patients.
The data reinforce CRENESSITY’s therapeutic profile and support its commercial strategy. The drug has already achieved a 10 % market penetration in the classic CAH population, and the two‑year results strengthen the evidence base that underpins future adoption. As the first‑in‑class therapy for a rare disease that has lacked new options for 70 years, the findings position Neurocrine to expand its share of the market and enhance long‑term patient outcomes.
Chief Medical Officer Sanjay Keswani said, “These two‑year findings showed that CRENESSITY achieved durable reductions in both androgen levels and glucocorticoid doses in children and adolescents with classic congenital adrenal hyperplasia, a population particularly vulnerable to the long‑term health impact of excess hormone exposure during growth and development. Improved hormonal control was associated with meaningful improvements in clinical outcomes, including body mass index and insulin resistance, supporting healthier outcomes as patients transition into adulthood.”
CRENESSITY received FDA approval on December 13 2024, and the CAHtalyst Pediatric study is the largest‑ever clinical trial program in classic CAH, enrolling 103 patients initially and following 86 through two years. The robust data set, combined with the company’s strong financial performance—total product sales exceeding $2.8 billion in 2025—underscores Neurocrine’s position as a leader in rare disease therapeutics and its capacity to translate clinical success into commercial growth.
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