FDA Approves Hydrocortisone Oral Solution for Adrenal Insufficiency

The US Food and Drug Administration (FDA) has approved hydrocortisone (KHINDIVI) oral solution as a replacement therapy in pediatric patients aged 5 years and older with adrenocortical insufficiency.1

Announced by Eton Pharmaceuticals on May 28, 2025, this approval marked the first FDA-approved oral solution formulation of hydrocortisone, designed to address administration and dosing accuracy in patients who may have difficulty swallowing pills or special administration needs.

“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, a professor in the division of endocrinology and vision of pediatric genetics and metabolism at the University of Minnesota.1 “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.”

Adrenal insufficiency is a rare, serious condition characterized by decreased production of aldosterone and cortisol due to reduced adrenal gland function. It can present acutely in an adrenal crisis or chronically, and if not recognized, can lead to very high morbidity and mortality.2 In the release, Eton Pharmaceuticals estimated that more than 5,000 adrenal insufficiency patients are present in the US between the ages of 5 and 17.1

The company currently commercializes the FDA–approved hydrocortisone (ALKINDI SPRINKLE) oral granules for pediatric patients with adrenocortical insufficiency.3 With this new approval, hydrocortisone oral solution can provide an additional option to meet the individual needs of both a patient and a caregiver.1

“For families facing the daily challenges of pediatric congenital adrenal hyperplasia (CAH), timely access to the right treatments is critical,” said Dina Matos, executive director of the CARES Foundation.1 “The introduction of [hydrocortisone oral solution] is a significant advancement, particularly because accurately splitting pills to achieve proper dosing for children has long been a struggle. The ability to dose patients more accurately is critical for treatment outcomes.”

Hydrocortisone oral solution will be available in a 1 mg/mL strength, offering simple, tailored dosing and eliminating the need to split or crush tablets. It will not require refrigeration, mixing, or shaking, offering a ready-to-use oral solution.

Eton Pharmaceuticals plans to make hydrocortisone oral solution available exclusively through Anovo, a specialty pharmacy serving patients with rare and chronic conditions. In the release, the company announced that hydrocortisone oral solution will be available to patients in the coming days.1

“For decades, patients have been seeking an FDA-approved hydrocortisone liquid that allows incremental, accurate dosing in the preferred dosage form for children,” said Sean Brynjelsen, chief executive officer of Eton Pharmaceuticals.1 “We are excited to now make it available to patients. Our commercial team is fully mobilized and ready to hit the ground running with the first week of approval.”

References

1. Eton Pharmaceuticals announces U.S. FDA approval for KhindiviTM (Hydrocortisone) Oral Solution. Eton Pharmaceuticals, Inc. May 28, 2025. Accessed May 29, 2025. https://ir.etonpharma.com/news-releases/news-release-details/eton-pharmaceuticals-announces-us-fda-approval-khindivitm.

2. Huecker MR, Bhutta BS, Dominique E. Adrenal Insufficiency. [Updated 2023 Aug 17]. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2025 Jan-. Available from: https://www.ncbi.nlm.nih.gov/books/NBK441832/

3. Eton Pharmaceuticals announces FDA approval of Orphan Drug alkindi® sprinkle (hydrocortisone) as replacement therapy in pediatric patients with adrenocortical insufficiency. Eton Pharmaceuticals, Inc. September 29, 2020. Accessed May 29, 2025. https://ir.etonpharma.com/news-releases/news-release-details/eton-pharmaceuticals-announces-fda-approval-orphan-drug-alkindir.