FDA Approves Hydrocortisone for Adrenocortical Insufficiency in Children


The orphan drug will be the first ever FDA-approved granular hydrocortisone formulation specifically developed for pediatric patients.

rare disease, FDA, AI

The US Food and Drug Administration (FDA) has approved hydrocortisone (ALKINDI SPRINKLE) oral granules as a replacement therapy for adrenocortical insufficiency (AI) in pediatric patients.

With the approval Eton Pharmaceuticals now has the only FDA-approved granular hydrocortisone formulation for the disease specifically designed for the use in children.

The orphan drug approval is based on the results of 6 clinical trials, including the first and only interventional phase 3 study of oral hydrocortisone for pediatric AI in neonates to children 8 years old.

Prior to the approval, oral hydrocortisone was only approved in tablet formulations of 5 mg and stronger. However, for pediatric patients, significantly lower doses are required, as well as the flexibility of precision titration. The new approval will allow the treatment to be available in 0.5 mg, 1 mg, 2 mg, and 5 mg strengths, giving doctors greater flexibility to individualize dosing based on each patient’s needs accordance with the instructions for dosage and administration.

Pediatric adrenocortical insufficiency (AI) is a rare disease characterized by an inability to synthesize and release cortisol, and sometimes aldosterone, causing excessive androgens.

“For years, we heard from parents about their struggle to provide the right dose to their child,” Dina M. Matos, Executive Director of the CARES Foundation, said in a statement. “We are thrilled the FDA has approved ALKINDI SPRINKLE for pediatric patients with Adrenocortical Insufficiency including patients with Congenital Adrenal Hyperplasia, a type of Adrenocortical Insufficiency.”

Eton expects the hydrocortisone treatment to be commercially available by the end of 2020.

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