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.