NEUROCRINE BIOSCIENCES, INC.

A mother raises funds for gene therapy for her son's rare disease, offering to name the treatment after the highest donor. Edgewise Therapeutics' experimental drug for Duchenne muscular dystrophy shows positive results. Neurocrine Biosciences gains FDA approval for a congenital adrenal hyperplasia drug. AbbVie acquires Nimble Therapeutics for $200 million. Viridian Therapeutics' drug improves thyroid eye disease symptoms. Novo Holdings' acquisition of Catalent is finalized. South African regulators end investigation into Vertex's cystic fibrosis drug Trikafta.

FDA approves Neurocrine’s Crenessity for CAH, an adjunct treatment to glucocorticoid replacement for controlling androgens in adults and children ages four and older. The approval is supported by the CAHtalyst clinical program, showing significant reductions in androstenedione and steroid doses. Crenessity is expected to be commercially available shortly.

Goldman Sachs raised Neurocrine Biosciences' price target to $182 from $177, maintaining a Buy rating. The FDA approved Neurocrine's drug Crenessity for classic congenital adrenal hyperplasia (CAH), marking a significant advancement in CAH treatment. Neurocrine's strong financial health, with 25.7% revenue growth and a current ratio of 4.37, supports its market position. The approval validates Neurocrine's drug development capabilities, enhancing its standing and reducing market risk. Goldman Sachs anticipates details on Crenessity's pricing strategy and evaluates launch progress through indicators like patient adoption and insurance coverage. Neurocrine's recent strong third-quarter earnings and a $300 million share repurchase plan further support its positive outlook.

The FDA approved Crenessity (crinecerfont) with glucocorticoids to control androgen levels in adults and pediatric patients (4+ years) with classic congenital adrenal hyperplasia (CAH). Crenessity reduces excessive adrenal androgen production, allowing for lower glucocorticoid doses. Trials showed significant reductions in glucocorticoid doses while maintaining androgen control. Crenessity carries warnings for acute adrenal insufficiency and potential drug interactions.

CRENESSITY™, approved by the FDA, is the first new treatment in 70 years for classic congenital adrenal hyperplasia (CAH), offering a novel approach to reduce adrenal androgens and glucocorticoid doses. Supported by the largest CAH clinical trial, it's available through PANTHERx Rare, with Neurocrine Access Support aiding patient access.

The FDA approved crinecerfont (Crenessity) as the first-in-class treatment for classic congenital adrenal hyperplasia (CAH) in patients 4 years and older. It reduces excessive adrenal androgen production, allowing for lower glucocorticoid doses. Two trials supported its approval, showing significant reductions in androstenedione levels and glucocorticoid doses. Common side effects include fatigue, dizziness, and arthralgia. It will be available in 50-mg and 100-mg capsules and a 50-mg/mL oral solution through PANTHERx Rare.

FDA approves Crenessity (crinecerfont) for classic congenital adrenal hyperplasia (CAH), reducing excess ACTH and adrenal androgen production, enabling glucocorticoid dose reduction. Available through PANTHERx Rare, with Neurocrine Access Support for patient assistance.

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, Inc.

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