Eton Pharmaceuticals, Inc., an innovative pharmaceutical company focused on developing and commercialising treatments for rare diseases, has announced the US Food and Drug Administration (FDA) approval of a New Drug Application (NDA) for KHINDIVI (hydrocortisone) Oral Solution as a replacement therapy in paediatric patients five years of age and older with adrenocortical insufficiency.

KHINDIVI is the only FDA-approved oral solution formulation of hydrocortisone. It comes in a 1mg/ml strength designed to eliminate the need to split or crush tablets, and to offer simple and accurate dosing specifically tailored to each patient’s needs. It does not require refrigeration, mixing, or shaking – it is a ready-to-use oral liquid solution.

KHINDIVI is designed to offer administration simplicity and dosing accuracy, and to provide a therapy option for patients who have difficulty swallowing tablets or with special administration needs, such as patients with a gastric tube.

“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. In addition, being in a position to commercialise KHINDIVI within days of this approval is a further testament to the executional excellence from our entire company,” said Sean Brynjelsen, CEO of Eton Pharmaceuticals.

He further added, “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 mobilised and ready to hit the ground running within the first week of approval. We’re committed to ensuring that paediatric endocrinologists across the country are aware of this important new treatment option.”

Dr. Kyriakie Sarafoglou, Professor, Division of Pediatric Endocrinology and Division of Pediatric Genetics and Metabolism, University of Minnesota said, “Managing adrenal insufficiency in paediatric patients requires precise and consistent hydrocortisone dosing that can be carefully titrated to small increments that address the individualised 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.”

Dina Matos, Executive Director of the CARES Foundation, a US-based organisation solely focused on the CAH community, said, “For families facing the daily challenges of paediatric 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.”

She further added, “Eton currently commercialises ALKINDI SPRINKLE (hydrocortisone) Oral Granules, which is FDA-approved for paediatric patients with adrenocortical insufficiency. The addition of KHINDIVI will provide adrenal insufficiency patients and caregivers with an additional option when choosing the treatment that best meets their individual needs.”

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 US between the ages of 5 and 17, and expects peak sales of KHINDIVI, combined with ALKINDI SPRINKLE, will exceed USD 50 million per year.

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. The program provides prescription fulfillment, insurance benefits investigation, educational support, financial assistance for qualified patients, and other services designed to help patients access treatment. Eton Cares will offer co-pay assistance to allow for $0 co-pays for qualifying patients.