Neurocrine Biosciences starts Phase 2 study of crinecerfont in children under 4 with classic CAH

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Neurocrine Biosciences has initiated a Phase 2 pediatric study of crinecerfont in children under 4 years with classic congenital adrenal hyperplasia in the United States, while also reaching target enrollment for a similar pediatric study in the European Union. Crinecerfont, also known as CRENESSITY, is already approved in the US for patients 4 years and older, and these new studies aim to test safety, dosing, and hormone control in the youngest patients where no approved therapies currently exist. The move could expand the addressable patient group and deepen the company's rare-disease franchise, though safety findings or dosing challenges in very young children could delay label expansion. Investors may watch for interim and final safety readouts, including adrenal crisis rates and glucocorticoid dosing needs, as well as potential regulatory steps toward a supplemental New Drug Application.

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Initiation of Phase 2 study in younger children could expand approved label and addressable patient group.

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