FDA Expands Indication of Cresemba for Use in Children With Invasive Aspergillosis, Invasive Mucormycosis

The FDA has approved an expanded indication to Astellas Pharma US Inc’s Cresemba (isavuconazonium sulfate) for use in pediatric patients for the treatment of invasive aspergillosis (IA) and invasive mucormycotic (IM). The regulatory action makes Cresemba the only azole antifungal treatment approved for use in pediatric patients as young as 1 year of age with AI and IM.

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“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,” Antonio C. Arrieta, MD, medical director, Pediatric Infectious Disease, Children's Health of Orange County, said 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 Cresemba for pediatric patients adds a much-needed treatment option.”

Cresemba will be available as an intravenous injection or capsule for fungal infections occurring in the blood or body. Adults and children aged 1 year and older are eligible for the IV formulation, whereas the prescribed capsule is only indicated for adults and children 6 years of age and older and who weigh 35 lbs or more.

In a press release, Astellas stated that it has yet to be determined whether Cresemba capsules are a safe and effective option for children who are under 6 years of age or who weigh less than 35 lbs. Further, it has yet to be determined whether the IV formulation is safe and effective for children younger than 1 year of age.

The FDA based its approval of Cresemba in part on findings from a Phase II open-label, non-comparative, multicenter study (NCT03816176). The researchers assessed the safety, efficacy, and pharmacokinetics of Cresemba for the treatment of IA or IM in 31 patients 1 through 17 years of age.

The study investigators administered 10 mg of Cresemba every eight hours on days one and two. After the first two days of treatment, patients with IA were administered treatment once-daily for up to 84 days, whereas patients with IM were administered treatment once daily for up to 180 days at a median treatment duration of 55 days. The study investigators observed that 54.8% of patients were able to achieve a successful response rate.

In terms of safety, the most common adverse effects (AEs) reported among both adults and children administered Cresemba were vomiting, diarrhea, and nausea. Additional AEs that were observed in adults only included headache, changes liver enzyme levels in the blood, low potassium, constipation, cough, swelling in the arms or legs, back pain, and shortness of breath. AEs that were only reported in children included stomach pain, rash, and itching.

The study investigators said that the approval expands the therapeutic options for children with IA or IM, which could potentially prevent 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,” Lynn Fenicchia, senior vice president and head of US Medical Specialties Business Unit, Astellas, said in the press release. “Patients are waiting, and we know that young [patients with IA and IM] are most at risk. [The] approval will bring hope to this highly vulnerable patient population with limited treatment options.”

Reference

FDA Approves Expanded Use of CRESEMBA® (isavuconazonium sulfate) in Children with Invasive Aspergillosis and Invasive Mucormycosis. Astellas. News release. December 8, 2023. Accessed December 11, 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.