FDA Sets PDUFA Date for Neffy Nasal Spray and Approves Crinecerfont for CAH Treatment
FDA Sets PDUFA Date for Neffy Nasal Spray
The FDA has established a Prescription Drug User Fee Act (PDUFA) target action date of March 6, 2025, for neffy, a 1 mg nasal spray intended for the treatment of anaphylaxis in children weighing 33 lbs or more. This decision follows the submission of a supplemental New Drug Application (sNDA) by the company, with product availability anticipated in the second quarter of 2025, pending approval. The initial approval of neffy was based on pharmacokinetic data showing comparable epinephrine blood concentrations to those achieved with approved epinephrine injection products. Clinical trials involving 175 healthy adults demonstrated that neffy produced similar increases in blood pressure and heart rate, essential effects for managing anaphylaxis.
Crinecerfont Approved for Congenital Adrenal Hyperplasia (CAH)
Crinecerfont, a novel therapeutic targeting corticotropin-releasing factor type 1 (CRF1) receptors, has been approved for the treatment of congenital adrenal hyperplasia (CAH). Clinical trials, including the CAHtalyst Pediatric Study (NCT04806451), have shown that crinecerfont can significantly reduce androstenedione and 17-OHP levels, with promising trends in body mass index, insulin resistance, and hirsutism. This approval marks a significant advancement in the management of CAH, offering a new approach to reduce excessive ACTH and androgen production while minimizing side effects.
Implications for Pediatric Care
These developments represent significant progress in pediatric care, offering new treatment options for life-threatening conditions like anaphylaxis and chronic conditions such as CAH. The approval of neffy and crinecerfont underscores the importance of innovative therapeutic approaches in improving patient outcomes and quality of life. As these treatments become available, healthcare providers will play a crucial role in integrating them into clinical practice, ensuring that patients receive the most effective and safe care possible.