Eton Pharmaceuticals announced on May 28, 2025, that the U.S. Food and Drug Administration has approved KHINDIVI (hydrocortisone) oral solution as replacement therapy for pediatric patients five years of age and older with adrenocortical insufficiency. The approval represents a significant milestone as KHINDIVI becomes the first and only FDA-approved oral solution formulation of hydrocortisone.
Addressing Critical Dosing Challenges
KHINDIVI comes in a 1mg/ml strength designed to eliminate the need to split or crush tablets, offering simple and accurate dosing specifically tailored to each patient's needs. The ready-to-use oral liquid solution requires no refrigeration, mixing, or shaking, providing a therapy option for patients who have difficulty swallowing tablets or with special administration needs, such as patients with a gastric tube.
"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, CEO of Eton Pharmaceuticals. "We are excited to now make it available to patients. Our commercial team is fully mobilized and ready to hit the ground running within the first week of approval."
Clinical Significance and Expert Perspectives
Dr. Kyriakie Sarafoglou, Professor in the Division of Pediatric Endocrinology & Division of Pediatric Genetics & Metabolism at the University of Minnesota, emphasized the clinical importance of the approval. "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," she said. "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."
The approval has been welcomed by patient advocacy groups. Dina Matos, Executive Director of the CARES Foundation—the only U.S. organization solely focused on the congenital adrenal hyperplasia (CAH) community—noted that "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."
Market Opportunity and Commercial Strategy
Adrenocortical insufficiency is a rare but serious condition in which the adrenal glands do not produce sufficient cortisol. Eton estimates that there are more than 5,000 adrenal insufficiency patients in the U.S. between the ages of 5 and 17. The company expects peak sales of KHINDIVI, combined with ALKINDI SPRINKLE, will exceed $50 million per year.
KHINDIVI will be promoted by Eton's existing team of pediatric endocrinology rare disease specialists. The company currently commercializes ALKINDI SPRINKLE (hydrocortisone) oral granules, which is FDA-approved for pediatric patients with adrenocortical insufficiency. The addition of KHINDIVI provides adrenal insufficiency patients and caregivers with an additional option when choosing treatment that best meets their individual needs.
Distribution and Access
KHINDIVI will be available in the coming days in the United States exclusively through Anovo, a specialty pharmacy dedicated to serving patients with rare and chronic conditions. Anovo will administer the Eton Cares Program in partnership with Eton Pharmaceuticals, providing prescription fulfillment, insurance benefits investigation, educational support, and financial assistance for qualified patients. The Eton Cares program will offer co-pay assistance to allow for $0 co-pays for qualifying patients.
Commercial launch is expected the week of June 2nd. Clinicians seeking to prescribe KHINDIVI can e-prescribe by selecting Anovo #5 or fax in a patient referral form to 855-813-2039.
Safety Profile and Limitations
KHINDIVI is indicated as replacement therapy in pediatric patients 5 years of age and older with adrenocortical insufficiency. However, it is not approved for increased dosing during periods of stress or acute events, requiring the use of a different hydrocortisone-containing drug product for stress dosing.
The drug carries several important warnings and precautions, including risks of adrenal crisis from undertreatment or sudden discontinuation, systemic adverse reactions due to inactive ingredients including hyperosmolarity and metabolic acidosis, and potential for immunosuppression with dosages greater than replacement therapy. The inactive ingredients polyethylene glycol 400, propylene glycol, and glycerin undergo substantial systemic absorption and may cause gastrointestinal irritation, particularly in pediatric patients less than 5 years of age.
Company Portfolio
Eton Pharmaceuticals is an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases. The company currently has eight commercial rare disease products: KHINDIVI, INCRELEX, ALKINDI SPRINKLE, GALZIN, PKU GOLIKE, Carglumic Acid, Betaine Anhydrous, and Nitisinone. The company has five additional product candidates in late-stage development: ET-600, Amglidia, ET-700, ET-800 and ZENEO hydrocortisone autoinjector.
