Eton Pharmaceuticals has received US regulatory approval for Khindivi, the first and only hydrocortisone oral solution cleared by the Food and Drug Administration (FDA), offering a simplified treatment option for paediatric patients with adrenal insufficiency.

Khindivi (hydrocortisone) oral solution has been approved as a replacement therapy for children aged five years and older living with adrenocortical insufficiency, a rare condition in which the adrenal glands fail to produce sufficient cortisol.

The formulation, available in a 1mg/ml concentration, is designed to support accurate dosing without the need to split or crush tablets. It does not require refrigeration, mixing, or shaking, and is intended for ease of administration in patients with swallowing difficulties or special needs, including those with gastric tubes.

Eton Pharmaceuticals expects to launch the product commercially in the US during the week of June 2. It anticipates peak combined annual sales of Khindivi and its existing hydrocortisone product, Alkindi Sprinkle, to exceed $50 million.

Sean Brynjelsen, CEO of Eton Pharmaceuticals, said the approval marks a significant step forward for the rare disease community.

He said:

“The FDA approval of Khindivi is a tremendous achievement for Eton and, more importantly, a pivotal step forward for paediatric patients with adrenal insufficiency. As a home-grown program, our team expertly navigated the development, clinical, and regulatory pathway.”

He added that the company’s commercial team is ready to begin distribution within the first week of approval, with a focus on raising awareness among paediatric endocrinologists.

The clinical importance of precise dosing in this patient group was underscored by Dr Kyriakie Sarafoglou, professor of pediatric endocrinology and genetics at the University of Minnesota.

He said: “Managing adrenal insufficiency in pediatric patients requires precise and consistent hydrocortisone dosing that can be carefully titrated to small increments. 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 launch was also welcomed by patient advocacy groups. Dina Matos, executive director of the CARES Foundation, said: “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.”

Khindivi will be distributed exclusively through Anovo, a specialty pharmacy serving patients with rare and chronic conditions. The Eton Cares program, operated in partnership with Anovo, will offer co-pay assistance and support services including benefits investigations and educational resources.

The addition of Khindivi to Eton’s portfolio provides another option alongside Alkindi Sprinkle for physicians and caregivers managing pediatric adrenal insufficiency.