Neurocrine Biosciences has announced the US Food and Drug Administration (FDA) approval of Crenessity (crinecerfont) capsules and oral solution.
Crenessity has been approved as an adjunct treatment to glucocorticoid replacement for controlling androgens in adults and children ages four and older with classic congenital adrenal hyperplasia (CAH).
Additionally, a Rare Pediatric Disease Priority Review Voucher was granted in connection with the approval of Crenessity.
In July this year, the FDA accepted Neurocrine Biosciences’ new drug applications (NDA) for crinecerfont and granted priority review.
Crinecerfont is a selective oral corticotropin-releasing factor type 1 receptor (CRF1) antagonist.
The FDA approval of Crenessity is supported by the results from the global CAHtalyst clinical programme which included the Phase 3 CAHtalyst Pediatric and CAHtalyst Adult studies.
The CAHtalyst Pediatric study demonstrated that Crenessity significantly reduced androstenedione levels in children with classic CAH, compared to placebo, with a substantial decrease by Week 4.
Patients on crinecerfont also achieved a significant reduction in glucocorticoid (GC) doses by Week 28, while maintaining or improving androgen levels.
The study showed that children taking the drug experienced approximately four times greater reductions in androstenedione and steroid doses compared to those on placebo.
Additionally, Crenessity treatment led to a 12-fold greater reduction in 17-hydroxyprogesterone levels compared to placebo.
The CAHtalyst Adult study showed that Crenessity met its primary endpoint, significantly reducing GC doses at Week 24 while maintaining or improving baseline androstenedione levels.
It also achieved the key secondary endpoint of reducing androstenedione levels at Week 4.
A significantly higher percentage of patients on Crenessity reached a GC dose in the physiologic range, with androstenedione levels maintained or improved, compared to only 18% in the placebo group.
Crinecerfont treatment led to approximately two times greater steroid dose reduction and eight times greater reduction in androstenedione compared to placebo.
Additionally, patients on Crenessity experienced a 37-times greater reduction in 17-hydroxyprogesterone (17-OHP) levels than those on placebo.
Neurocrine Biosciences CEO Kyle Gano said: “The approval of Crenessity is a significant milestone for the CAH community, and we are grateful to the individuals who participated in our clinical trials, including their families and caregivers, and to the clinical investigators who helped advance a new therapy and class of medicines.”
Crinecerfont is expected to be commercially available shortly. The medication will be distributed through PANTHERx Rare, a speciality pharmacy.
