FDA Approves Khindivi, First Oral Solution of Hydrocortisone, for Pediatric Patients

Updated Thursday, May 29, 2025, at 10:47 AM.

The FDA approved the new drug application (NDA) for hydrocortisone oral solution (formerly ET-400, Khindivi; Eton Pharmaceuticals) as a replacement therapy in pediatric patients with adrenocortical insufficiency who are 5 years of age and older. This decision is the first oral solution formulation of hydrocortisone to be FDA-approved, according to a news release from the manufacturers.^1,2

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“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,” Kyriakie Sarafoglou, MD, professor, Division of Pediatric Endocrinology & Division of Pediatric Genetics & Metabolism, University of Minnesota, said in a news release. “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.”¹

Khindivi comes in a 1-mg per ml strength and is designed to provide treatment for patients without the need to split or crush tablets. Additionally, the oral formulation offers a simple and accurate dosing that can be specifically tailored to an individual patient’s needs. It is a ready-to-use solution that does not require refrigeration, mixing, or shaking. It is also an option for patients who have difficulty swallowing tablets or who have special administration needs. The NDA for the oral formulation of hydrocortisone was accepted by the FDA on July 15, 2024, with a Prescription Drug User Fee Act target action date of February 28, 2025.^1,2

“For families facing the daily challenges of pediatric congenital adrenal hyperplasia, timely access to the right treatments is critical,” Dina Matos, executive director of the CARES Foundation, said in the news release. “The introduction of Khindivi 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… This marks meaningful progress for our community and a vital step toward easing the daily burden on parents and caregivers.”¹

Adrenocortical insufficiency, or Addison disease, is a rare but serious condition in which the adrenal glands do not produce sufficient cortisol, according to the news release. There are over 5000 patients with adrenal insufficiency between the ages of 5 and 17 in the US. According to Johns Hopkins Medicine, the condition occurs when the adrenal glands are unable to create enough of the hormone cortisol, which is essential in helping the body break down fats, proteins, and carbohydrates, as well as control blood pressure.^1,3

The condition is caused when the immune system mistakenly attacks the healthy adrenal glands; however, other causes may be cancers, fungal infections, tuberculosis infection of the adrenal glands, or inherited disorders of the endocrine glands. Common symptoms include fatigue, weakness, dizziness, weight loss, dehydration, low blood pressure and sugar levels, gastrointestinal complications (eg, nausea, vomiting, diarrhea), and, in women, irregular or no menstruation.³

“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. “We’re committed to ensuring that pediatric endocrinologists across the country are aware of this important new treatment option.”¹

REFERENCES

1. Eton Pharmaceuticals. Eton Pharmaceuticals Announces U.S. FDA Approval for KHINDIVI™ (hydrocortisone) Oral Solution. News 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. Eton Pharmaceuticals Announces FDA Acceptance of New Drug Application for ET-400 (Hydrocortisone Oral Solution). News release. July 15, 2024. Accessed May 29, 2025. https://ir.etonpharma.com/news-releases/news-release-details/eton-pharmaceuticals-announces-fda-acceptance-new-drug-0

3. Johns Hopkins Medicine. Adrenal Insufficiency (Addison's Disease). Accessed May 29, 2025. https://www.hopkinsmedicine.org/health/conditions-and-diseases/underactive-adrenal-glands--addisons-disease