Tildacerfont

Neurocrine Biosciences' crinecerfont, targeting classic congenital adrenal hyperplasia (CAH), receives FDA Priority Review. If approved, it would be the first new CAH treatment in 70 years, offering a novel mechanism. CAH, caused by 21-hydroxylase gene mutations, affects hormone production in adrenal glands, with the US having the highest prevalence. Current treatments focus on managing symptoms, with EFMODY as the only approved drug in Europe. Emerging therapies like crinecerfont aim to normalize hormone levels and address the root cause of CAH.