FDA Approves Neurocrine’s Crenessity for Classic Congenital Adrenal Hyperplasia
FDA Approves First-Ever Treatment for Classic Congenital Adrenal Hyperplasia
San Diego-based Neurocrine Biosciences has secured a groundbreaking FDA approval for Crenessity (crinecerfont), a first-in-class oral medication for classic congenital adrenal hyperplasia (CAH). The approval, granted nearly two weeks ahead of schedule, marks a important advancement in treating this rare genetic disorder, which has seen no new treatment options in over 70 years.
Crenessity,a selective corticotropin-releasing factor type 1 receptor (CRF) antagonist,offers a new hope for the estimated 30,000 Americans living with CAH. The twice-daily oral medication works by reducing excess adrenocorticotropic hormone (ACTH) and downstream androgen production, allowing for a reduction in glucocorticoid doses.
“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,” said Dina matos, executive director of the CARES Foundation, in a statement.Long-Term Relief from Steroid Side Effects
For decades, glucocorticoid replacement therapy has been the mainstay treatment for CAH.Though, long-term use of high doses can lead to serious side effects, including weight gain, heart problems, and bone loss.Crenessity offers a potential solution by allowing for glucocorticoid dose reduction while maintaining androgen control.
The FDA approval was supported by positive results from two phase 3 clinical trials, one in children and adolescents and another in adults. Both studies demonstrated that crinecerfont substantially reduced daily glucocorticoid doses compared to placebo while effectively controlling androgen levels.
Neurocrine’s Third Approved Drug
Crenessity is Neurocrine’s third approved drug. The company’s blockbuster drug Ingrezza, approved in 2017 for tardive dyskinesia, recently received a label expansion for chorea in patients with Huntington’s disease.Orilissa, an endometriosis drug approved in 2018, is licensed to AbbVie.
Neurocrine anticipates making Crenessity available to patients by next week. Pricing information has not yet been released. Earlier this year,the company launched an educational campaign titled “whatthec@H?!” to raise awareness and build a community for CAH patients.This FDA approval represents a major milestone for the CAH community, offering a new treatment option with the potential to significantly improve the lives of patients and their families.
A Breakthrough for CAH: FDA Approves First-Ever Treatment
The FDA has just granted accelerated approval to Crenessity (crinecerfont),marking a historic moment for the 30,000 Americans living with classic congenital adrenal hyperplasia (CAH). Developed by San Diego-based Neurocrine Biosciences, Crenessity is the first-ever oral medication specifically designed to treat this rare genetic disorder.
For decades, the only treatment available for CAH has been high-dose glucocorticoid therapy. While effective in managing symptoms, long-term use can lead to important side effects including weight gain, heart problems, and bone loss, significantly impacting patients’ quality of life.
“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,” said Dina Matos, executive director of the CARES Foundation.
Crenessity works by targeting the underlying cause of CAH. As a selective corticotropin-releasing factor type 1 receptor (CRF) antagonist, it reduces excess adrenocorticotropic hormone (ACTH) and downstream androgen production. This allows for a reduction in glucocorticoid doses while still maintaining control over androgen levels.
The FDA’s approval is based on the positive results of two Phase 3 clinical trials, both demonstrating Crenessity’s ability to significantly reduce daily glucocorticoid doses compared to placebo while effectively controlling androgen levels in both children, adolescents, and adults with CAH.
This approval represents a major milestone, offering a new treatment option with the potential to significantly improve the lives of CAH patients and their families. Neurocrine anticipates making Crenessity available to patients by next week with pricing information to be released soon.