Eton Pharma Receives U.S. FDA Approval For KHINDIVI™ (Hydrocortisone) Oral Solution

Eton Pharmaceuticals, a company focused on developing and commercializing treatments for rare diseases, has announced that the U.S. Food and Drug Administration (FDA) has approved a New Drug Application (NDA) for KHINDIVI (hydrocortisone) Oral Solution. The medication is now approved as a replacement therapy for pediatric patients aged five years and older who are living with adrenocortical insufficiency, a rare and potentially life-threatening condition in which the adrenal glands fail to produce enough cortisol.

KHINDIVI is the first and only FDA-approved oral liquid formulation of hydrocortisone. It is available in a 1 mg/ml concentration, specifically developed to make dosing easier and more precise, especially for children who may have difficulty swallowing tablets or require alternative methods of administration, such as through a gastric tube. Unlike traditional tablet forms that may require splitting or crushing, KHINDIVI comes ready to use and does not require mixing, shaking, or refrigeration. These features make the treatment simpler and more convenient for both patients and caregivers.

Sean Brynjelsen, CEO of Eton Pharmaceuticals, said in a statement, “The FDA approval of KHINDIVI is a tremendous achievement for Eton and more importantly, a pivotal step forward for pediatric patients with adrenal insufficiency. As a home-grown program, our team expertly navigated the development, clinical and regulatory pathway. In addition, being in a position to commercialize KHINDIVI within days of this approval is a further testament to the executional excellence from our entire company,” 

He further continued, “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. 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.”

The medication will be promoted by Eton Pharmaceuticals’ existing team of pediatric endocrinology specialists. Eton already markets ALKINDI SPRINKLE, an FDA-approved oral granule form of hydrocortisone for younger pediatric patients with the same condition. The approval of KHINDIVI expands Eton’s portfolio, providing another important option for families managing adrenal insufficiency. The availability of both KHINDIVI and ALKINDI SPRINKLE offers physicians and caregivers greater flexibility in selecting treatments that best match the individual needs of patients.

Adrenocortical insufficiency affects a relatively small patient population in the United States, with Eton estimating more than 5,000 affected individuals between the ages of five and seventeen. Despite being a rare disease, the market demand is significant, and the company expects that the combined peak sales of KHINDIVI and ALKINDI SPRINKLE could exceed $50 million annually.

Dr. Kyriakie Sarafoglou, Professor, Division of Pediatric Endocrinology & Division of Pediatric Genetics & Metabolism, University of Minnesota, stated, “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. 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.”

“For families facing the daily challenges of pediatric congenital adrenal hyperplasia (CAH), timely access to the right treatments is critical. 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. We commend Eton for working to make this therapy accessible through specialty channels. This marks meaningful progress for our community and a vital step toward easing the daily burden on parents and caregivers,” commented Dina Matos, Executive Director of the CARES Foundation, the only U.S. organization solely focused on the CAH community. 

KHINDIVI will be made available in the U.S. exclusively through Anovo, a specialty pharmacy that focuses on serving patients with rare and chronic conditions. Anovo will also manage the Eton Cares Program in partnership with Eton Pharmaceuticals. This program is designed to support patients throughout the treatment process, offering services such as prescription fulfillment, insurance benefit verification, educational resources, and financial assistance for eligible patients. For those who qualify, the program will include co-pay assistance, making it possible to access the medication with zero out-of-pocket costs.