FDA Approves Therapy for Fungal Infections in Young Children


The approval of the antifungal, isavuconazonium sulfate (Cresemba), is indicated for children as young as 1 year of age and the approval was based from 2 clinical trials.

On Friday, the FDA announced it had approved the antifungal therapy isavuconazonium sulfate (Cresemba). The therapy is indicated for the treatment of invasive aspergillosis (IA) and invasive mucormycosis (IM) in pediatric patients 1 year of age and older.1 In the youngest patient population, isavuconazonium sulfate is recommended as an injection. In addition, isavuconazonium sulfate capsules are now approved for pediatric patients 6 years of age and older who weigh 35 pounds and greater. The capsules had already been approved for adults.1

With yesterday’s FDA approval, this becomes the only azole antifungal therapy available for the treatment of IA and IM in patients as young as 1 years old.1

"Patients are waiting, and we know that young IA and IM patients are most at risk. Today's approval will bring hope to this highly vulnerable patient population with limited treatment options," Lynn Fenicchia, senior vice president and Head of US Medical Specialties Business Unit, Astellas, said in a statement.1

For pediatric patients with compromised immune systems, they are at a higher risk for IA and IM. "Invasive fungal infections are a significant concern to healthcare professionals who care for pediatric, adolescents and young adults with hematologic malignancies, transplant recipients and primary immune deficiencies," said Antonio Arrieta, MD, medical director, Pediatric Infectious Disease, Children's Health of Orange County. "IA, currently the most common invasive mold infection in these patients, and IM are associated with significant morbidity and high fatality rates. The FDA approval of Cresemba for pediatric patients adds a much-needed treatment option."1

What the Data Shows

Isavuconazonium sulfate is manufactured by Astellas Pharma. Back in 2010, Basilea Pharmaceutica partnered with Astellas to complete phase 3 trials, obtain regulatory approvals, and market the drug. In 2013 and 2014, the partners won orphan drug designation in the US for isavuconazonium for treating invasive aspergillosis, mucormycosis, and invasive candidiasis.2

Isavuconazonium is an azole antifungal, and the approval is based off the results of 2 clinical trials, including a phase 2 open-label, non-comparative, multicenter study evaluating the safety, efficacy and pharmacokinetics of isavuconazonium sulfate for the treatment of IA or IM in pediatric patients aged 1 to 17 years old. In the study, 31 patients aged 1 to 17 with possible, probable or proven IA or IM were treated with isavuconazonium sulfate (10 mg/kg) every 8 hours on days 1 and 2, and once-daily thereafter for ≤84 days for IA or ≤180 days for IM.

Median treatment duration was 55 days. All-cause case fatality through Day 42 was 6.5% and through day 84 was 9.7% which occurred during the follow-up period and none were considered treatment-related. Successful response rates were 54.8% at the end of treatment. Treatment emergent adverse events (TEAEs) occurred in 93.5% of patients and 29.0% patients experienced drug-related TEAEs. Treatment was withdrawn in two patients due to TEAEs. Serious TEAEs occurred in 58.1% of patients and were assessed as drug-related by the investigator in 1 patient.


1. FDA Approves Expanded Use of CRESEMBA (isavuconazonium sulfate) in Children with Invasive Aspergillosis and Invasive Mucormycosis. Astellas Pharma press release. December 8, 2023. https://newsroom.astellas.us/2023-12-08-FDA-Approves-Expanded-Use-of-CRESEMBA-R-isavuconazonium-sulfate-in-Children-with-Invasive-Aspergillosis-and-Invasive-Mucormycosis

2. Isavuconazonium. Wikipedia. Accessed December 9, 2023. https://en.wikipedia.org/wiki/Isavuconazonium

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