FDA approves hydrocortisone oral solution for children 5 years, older with adrenocortical insufficiency

FDA approves hydrocortisone oral solution for children 5 years, older with adrenocortical insufficiency | Image credit: Contemporary Pediatrics

On May 28, 2025, the FDA approved Eton Pharmaceuticals' new drug application (NDA) for hydrocortisone oral solution (Khindivi) as a replacement therapy for pediatric patients aged 5 years or older with adrenocortical insufficiency, according to a press release from the company.¹

Approval of the NDA makes the therapy the only FDA-approved oral solution of hydrocortisone. The therapy comes in a 1 mg/ml strength and is designed to eliminate the need to split or crush tablets and to offer simple and accurate dosing specifically tailored to each patient's needs. Eton stated that the oral therapy does not require refrigeration, mixing, or shaking, and that it is a ready-to-use solution.¹ The FDA accepted the NDA for the oral solution, formally named ET-400, on July 15, 2024.²

"Managing adrenal insufficiency in pediatric patients requires precise and consistent hydrocortisone dosing that can be carefully titrated to small increments that address the individualized pharmacokinetic needs of each child,” said Kyriakie Sarafoglou, MD, professor, Division of Pediatric Endocrinology & Division of Pediatric Genetics & Metabolism, University of Minnesota. "The availability of an FDA-approved oral hydrocortisone liquid solution offers physicians a new tool to dose patients accurately, which is important to clinical outcomes during this dynamic period of growth and development," said Sarafoglou in a statement.¹

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Adrenocortical insufficiency is a rare, serious condition in which adrenal glands do not produce sufficient cortisol, affecting more than 5000 patients in the United States between the ages of 5 and 17 years, according to Eton estimates.

“For decades, patients have been seeking an FDA-approved hydrocortisone liquid that allows incremental, accurate dosing in the preferred dosage form for children. We are excited to now make it available to patients," said Sean Brynjelsen, CEO of Eton Pharmaceuticals, in the press release. "Our commercial team is fully mobilized and ready to hit the ground running within the first week of approval. We’re committed to ensuring that pediatric endocrinologists across the country are aware of this important new treatment option."

Eton stated that the hydrocortisone oral solution will be available in the United States in the coming days, exclusively through Anovo, a rare and chronic conditions specialty pharmacy. The pharmacy will administer the Eton Cares Program, which provides prescription fulfillment, insurance benefits investigation, educational support, financial assistance for qualified patients, and other services designed to help patients access treatment, according to the company.

Hydrocortisone oral solution is not approved for increased dosing during periods of stress or acute events and has a contraindication of hypersensitivity to hydrocortisone or any of the other ingredients in the newly-approved therapy.

References:

1. Eton Pharmaceuticals announces US FDA approval for Khindivi (hydrocortisone) oral solution. Eton Pharmaceuticals. Press release. May 28, 2025. Accessed May 29, 2025. https://ir.etonpharma.com/news-releases/news-release-details/eton-pharmaceuticals-announces-us-fda-approval-khindivitm

2. Eton Pharmaceuticals announces FDA acceptance of new drug application for ET-400 (hydrocortisone oral solution). Press release. July 15, 2025. Accessed May 29, 2025. https://ir.etonpharma.com/news-releases/news-release-details/eton-pharmaceuticals-announces-fda-acceptance-new-drug-0