Use of Expanded Access Fosmanogepix in Two Pediatric Patients With Rhino-Orbital-Cerebral Mucormycosis.

Mucormycosis is a severe fungal infection with high morbidity and mortality. Pharmacotherapeutic options are limited, often used in combination, and associated with significant adverse effects. Fosmanogepix is an investigational antifungal with a novel mechanism of action and has shown broad in vitro activity. Early clinical trials suggest a manageable adverse effect profile, making it an attractive treatment option. Two pediatric patients presented in diabetic ketoacidosis and were found to have extensive rhino-orbital-cerebral mucormycosis. Treatment with standard of care, including liposomal amphotericin B, micafungin, and azole antifungals along with nonpharmacologic measures, was unable to eradicate the infection. Expanded access fosmanogepix was added to the multimodal treatment regimens. Each patient improved and more poorly tolerated antifungals were able to be discontinued. Both patients completed several months of fosmanogepix, tolerated therapy well, and survived their deadly infections.