FDA grants priority review to crinecerfont for treatment of congenital adrenal hyperplasia
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Key takeaways:
- The FDA set target action dates for December to review two formulations of crinecerfont to treat CAH.
- The oral therapy met primary endpoints in an adult trial and a pediatric trial published in June.
The FDA has granted priority review designation for two new drug applications for a selective corticotropin-releasing factor type 1 receptor to treat congenital adrenal hyperplasia, according to a press release.
As Healio previously reported, data from two trials showed crinecerfont (Neurocrine Biosciences) reduced the need for glucocorticoids compared with placebo among adults with congenital adrenal hyperplasia (CAH) and was tied to greater reductions in androstenedione levels than placebo for children with the disorder. Following the presentation of data from both trials at the ENDO annual meeting and simultaneous publication in The New England Journal of Medicine, the FDA accepted two NDAs with priority review designations for crinecerfont to treat CAH among children, adolescents and adults.
The two NDAs will review the primary presentation of efficacy and safety of crinecerfont for the treatment of CAH, with one NDA reviewing a capsule formulation and the second an oral solution formulation. The Prescription Drug User Fee Act target action date is Dec. 29 for the capsule formulation and Dec. 30 for the oral solution formulation. The FDA is not planning to hold an advisory committee meeting to discuss the applications, according to the press release.
“Receipt of a priority review reflects the FDA’s agreement that CAH is a serious condition and there is an urgent need for patients to have access to new treatments,” Eiry W. Roberts, MD, chief medical officer at Neurocrine Biosciences, said in a press release. “Crinecerfont’s compelling efficacy results and excellent safety profile support our filing, and we look forward to working with the FDA as we head toward the [Prescription Drug User Fee Act] dates at the end of 2024.”
Crinecerfont previously receiving orphan drug designation from the FDA in March 2019. As Healio previously reported, the FDA granted breakthrough therapy designation to the medication in December 2023.
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