Crinecerfont

The FDA approved Crinecerfont for classic congenital adrenal hyperplasia, reducing glucocorticoid doses while maintaining androstenedione control in patients aged 4 and older. Trials showed Crinecerfont reduced glucocorticoid doses by 27% in adults and 18% in children while controlling androstenedione levels. It carries warnings for acute adrenal insufficiency and should not be used by those with hypersensitivity to its components.

Neurocrine Biosciences announces FDA approval of CRENESSITY for classic congenital adrenal hyperplasia, reducing ACTH and adrenal androgen production, available via PANTHERx Rare pharmacy.

FDA approves CRENESSITY™, a first-in-class treatment for classic congenital adrenal hyperplasia (CAH), reducing excess adrenal androgens and enabling glucocorticoid dose reduction. Supported by the largest CAH clinical trial program, CRENESSITY is expected to be commercially available soon, with Neurocrine Access Support aiding patient access.

The FDA approved Neurocrine Biosciences' crinecerfont (Crenessity) for congenital adrenal hyperplasia (CAH) in adults and children aged 4+ to reduce glucocorticoid doses while maintaining androstenedione control. Two trials showed crinecerfont significantly reduced glucocorticoid doses and serum androstenedione levels compared to placebo. Common side effects include fatigue, dizziness, and headache. The drug carries a warning for acute adrenal insufficiency.

The FDA is set to decide on ten drug applications, including Ionis' olezarsen for familial chylomicronemia syndrome, Lexicon's sotagliflozin for type 1 diabetes, AstraZeneca and Daiichi Sankyo's Dato-DXd for non-squamous NSCLC, Zealand Pharma's glepaglutide for short bowel syndrome, Rhythm Pharmaceuticals' Imcivree for genetic obesity in children, Checkpoint Therapeutics' cosibelimab for cutaneous squamous cell carcinoma, Mirum's chenodiol for cerebrotendinous xanthomatosis, Bristol Myers Squibb's subcutaneous Opdivo, and Neurocrine's crinecerfont for congenital adrenal hyperplasia.

FDA decisions in December include BeiGene's Tevimbra for gastric cancer, Ionis Pharma's Olezarsen for familial chylomicronemia syndrome, Lexicon Pharma's Zynquista for type 1 diabetes, AstraZeneca's Datopotamab deruxtecan for lung cancer, Zealand Pharma's Glepaglutide for short bowel syndrome, Rhythm Pharma's IMCIVREE for obesity in children, Soleno's DCCR for Prader-Willi syndrome, Checkpoint's Cosibelimab for skin cancer, Mirum's Chenodiol for cerebrotendinous xanthomatosis, Bristol-Myers' subcutaneous Opdivo, and Neurocrine's Crinecerfont for congenital adrenal hyperplasia.

Neurocrine Biosciences reported $622.1M in Q3 2024 revenue, driven by INGREZZA sales, and achieved milestones like FDA Priority Review for crinecerfont. Challenges include commercialization risks and competitive pressures.