After seven decades with no advances in the treatment of the genetic disorder classic congenital adrenal hyperplasia (CAH), Neurocrine Biosciences has scored FDA approval for its first-in-class, twice-daily oral medicine Crenessity (crinecerfont).
The blessing came nearly two weeks before its FDA target date and is termed as “paradigm shifting” by the San Diego-based company. The selective oral corticotropin-releasing factor type 1 receptor (CRF) antagonist can be used by CAH patients age 4 and older.
Serving as an add-on to glucocorticoid replacement therapies, Crenessity reduces excess adrenocorticotropic hormone (ACTH) and downstream adrenal androgen production, allowing for glucocorticoid dose reduction.
With the approval, Neurocrine's share price increased by 6%.
It’s a key breakthrough for CAH patients as long-term use of glucocorticoid replacement therapies at high doses can cause weight gain, heart problems and bone loss.
“Patients and families struggle to achieve balance between managing the symptoms of CAH and the side effects or complications of treatment with high-dose steroids, which may impact quality of life,” Dina Matos, the executive director of the CARES Foundation, said in a Friday release facilitated by Neurocrine.
CAH is an imbalance between cortisol and androgen hormones. When the body can’t make enough of the cortisol hormone, another hormone known as ACTH climbs. This causes overproduction of androgens, or male sex hormones like testosterone. It is estimated that there are 30,000 with the disorder in the U.S.
The company expects to make Crenessity available by next week. Neurocrine added in an email that it will reveal the price of Crenessity in the coming days. Earlier this year, the company launched an educational campaign titled WhattheC@H?! to help build a community of patients with CAH.
The nod is accompanied by a valuable rare pediatric disease priority review voucher, which can be used to expedite the FDA's assessment of another application. The company said it has not decided whether it will sell or hold onto its PRV.
Crenessity has been tested in two separate phase 3 studies: one for children and adolescents, and the other for adults. Results of the former study, which were released in 2023, showed that after 28 weeks, crinecerfont hit its primary endpoint of producing a statistically significant reduction in daily glucocorticoid dose compared to placebo, while also maintaining androgen control. Those findings echoed similar results from the adult-focused trial that were unveiled a month earlier.
This is the third approved drug for Neurocrine. Ingrezza was approved for tardive dyskinesia in 2017 and won a key label expansion in 2023 to treat chorea in patients with Huntington’s disease. The blockbuster generated sales of $613 million in the third quarter and is expected to rack up revenue of $2.3 billion in 2024.
The company also won a 2018 approval for endometriosis drug Orilissa, which it licenses to AbbVie.