VIDEO: Possible game-changer for people with congenital adrenal hyperplasia
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BOSTON —In this video exclusive, Richard J. Auchus, MD, PhD, describes a possible new treatment paradigm for congenital adrenal hyperplasia with crinecerfont, an investigational drug granted a breakthrough designation in December.
At ENDO 2024, researchers presented data from the adult and pediatric CAHtalyst phase 3 trials, which compared crinecerfont (Neurocrine Biosciences), an oral selective corticotropin-releasing factor type 1 receptor, with placebo for people with CAH. As Healio previously reported, crinecerfont was tied to a lower daily glucocorticoid dose with maintenance of androgen control than placebo at 24 weeks for adults and at 28 weeks for children. Crinecerfont was also associated with greater reductions in androstenedione levels than placebo at 4 weeks in the pediatric trial.
Auchus, who is professor of internal medicine and pharmacology at the University of Michigan and an investigator on both CAHtalyst trials, said crinecerfont offers people with CAH a potential alternative to glucocorticoids for maintaining androgen control. He described the drug as a potential game-changer if it receives approval from the FDA.
“When people stay in control during childhood and adolescence, they tend to be easier to control in adulthood too, which we hope will spare people the complications of this disease,” Auchus said.
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