The approval marks isavuconazonium sulfate as the only granted azole antifungal therapy for pediatric patients with invasive aspergillosia and invasive mucormycotic who are as young as age 1.
The FDA has approved isavuconazonium sulfate (Cresemba, Astellas Pharma US Inc), to treat invasive aspergillosis (IA) and invasive mucormycotic (IM) in pediatric patients. The approval marks isavuconazonium sulfate as the only granted azole antifungal therapy for pediatric individuals with AI and IM that are as young as 1 year old.
“Invasive fungal infections are a significant concern to health care professionals who care for pediatric, adolescents, and young adults with hematologic malignancies, transplant recipients, and primary immune deficiencies,” said Antonio C. Arrieta, MD, medical director, Pediatric Infectious Disease, Children's Health of Orange County, in a press release. “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 [isavuconazonium sulfate] for pediatric patients adds a much-needed treatment option.”
Isavuconazonium sulfate is aimed to treat fungal infections that occur in the blood or body through an injection or capsule. The study authors noted that the prescribed injection is eligible to treat both adults and children that are age 1 and older; however, the prescribed capsule can only treat adults and children that are age 6 years and older who weigh 35 pounds or more.
The study authors noted that is it not yet known if isavuconazonium sulfate capsules are a safe and effective form of treatment for children who are either less than age 6 years or age 6 years but weigh less than 35 pounds. Additionally, researchers are also unaware if the infection is safe and effective for children younger than age 1.
The approval of isavuconazonium sulfate was based on 2 pediatric clinical studies—one was a phase 2 open-label, non-comparative, multicenter study (NCT03816176). The researchers assessed the safety, efficacy, and pharmacokinetics of isavuconazonium sulfate to treat IA or IM in 31 individuals who were age 1 to 17 years.
According to the study, the individuals received 10 mg of isavuconazonium sulfate, every 8 hours on days 1 and 2. Following days 1 and 2, the individuals with IA received treatment once-daily up to 84 days and individuals with IM received treatment once daily for up to 180 days; however, the median treatment duration was 55 days.
The study authors noted that the results showed that 54.8% of individuals reached a successful response rate. The most common adverse effects (AEs) among adults and children was vomiting, diarrhea, and nausea. Other AEs that occurred only in adults included headache, changes in level of liver enzyme in blood, low potassium, constipation, cough, swelling of arms or legs, back pain, and shortness of breath. AEs that occurred only in children included stomach pain, rash, and itching.
The study authors noted that the approval expands treatment options for children that experience IA or IM, preventing potentially fatal infections.
“The recent COVID-19 pandemic drove an uptick in these serious and life-threatening fungal infections, presenting an even greater urgency to address this growing unmet medical need,” said Lynn Fenicchia, senior vice president and head of US Medical Specialties Business Unit, Astellas, in a press release. “Patients are waiting, and we know that young [patients with IA and IM] are most at risk. Today’s approval will bring hope to this highly vulnerable patient population with limited treatment options.”
FDA Approves Expanded Use of CRESEMBA® (isavuconazonium sulfate) in Children with Invasive Aspergillosis and Invasive Mucormycosis. Astellas. News release. December 8, 2023. Accessed 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.