Eton Pharmaceuticals announced positive results from its pivotal bioequivalence study for ET-600, a proprietary oral solution formulation of desmopressin developed for treating central diabetes insipidus. The study demonstrated pharmacokinetic equivalence to the FDA-approved reference product, setting the stage for a New Drug Application (NDA) submission in April 2025.
The bioequivalence trial enrolled 75 healthy adult subjects who were randomly assigned to receive both the test and reference drugs in an open-label, balanced, randomized, single-dose, three-treatment, three-sequence, three-period, three-way crossover design. The successful results position ET-600 to potentially become the only FDA-approved oral liquid formulation of desmopressin in the United States.
"We are pleased to see ET-600 pass its pivotal study and move one step closer to reaching patients," said Sean Brynjelsen, CEO of Eton Pharmaceuticals. "Through our deep relationships in the pediatric endocrinology community, we've come to appreciate the significant need for a desmopressin medication that can accommodate the precise and titratable doses necessary for pediatric patients."
Addressing an Unmet Need in Pediatric Endocrinology
Central diabetes insipidus, a rare condition affecting approximately 3,000 pediatric patients in the United States, results from insufficient production of antidiuretic hormone (vasopressin) by the pituitary gland. This leads to excessive urination and thirst, which can be particularly challenging to manage in children.
The development of ET-600 addresses a critical gap in treatment options for pediatric patients who require precise dosing adjustments. Current tablet formulations of desmopressin present challenges for pediatric dosing, where flexibility and accuracy are essential for effective management of the condition.
Patent Protection and Commercial Strategy
Eton has secured intellectual property protection for ET-600's formulation with a patent extending to 2044, with an additional patent application under review by the United States Patent and Trademark Office. This long-term exclusivity provides a strong foundation for the company's commercial strategy.
The company has already initiated pre-launch commercial readiness activities in anticipation of a potential market launch in the first quarter of 2026, pending FDA approval. Eton's established presence in the rare disease space, with seven commercial products already in its portfolio, positions it well to introduce ET-600 to the pediatric endocrinology community.
Expanding Rare Disease Portfolio
ET-600 represents an important addition to Eton's growing rare disease portfolio, which currently includes INCRELEX®, ALKINDI SPRINKLE®, GALZIN®, PKU GOLIKE®, Carglumic Acid, Betaine Anhydrous, and Nitisinone. The company also has three other product candidates in late-stage development: ET-400, Amglidia®, and ZENEO® hydrocortisone autoinjector.
The successful development of ET-600 aligns with Eton's strategic focus on addressing unmet needs in rare diseases, particularly those affecting pediatric populations where specialized formulations can significantly improve treatment outcomes and quality of life.
If approved, ET-600 would provide endocrinologists and pediatricians with a valuable new option for managing central diabetes insipidus in their youngest patients, offering the precision dosing capabilities that current formulations lack.
