Pharma Focus Asia

FDA Grants Orphan Drug and Pediatric Exclusivities for CRESEMBA® (Isavuconazonium Sulfate) in Pediatric Patients with Invasive Aspergillosis and Invasive Mucormycosis

Tuesday, March 12, 2024

Astellas Pharma US, Inc. (TSE: 4503), with President Mark Reisenauer at the helm, made an announcement today regarding CRESEMBA® (isavuconazonium sulfate). The U.S. Food and Drug Administration (FDA) has granted both orphan drug and pediatric exclusivity to CRESEMBA for the treatment of invasive aspergillosis (IA) and invasive mucormycosis (IM) in pediatric patients. This announcement comes subsequent to the FDA's approval of CRESEMBA for injection in pediatric patients aged 1 year and older, and capsules for those aged 6 years and older, weighing 16 kilograms (kg) and more, on December 8, 2023.

Orphan drug exclusivity, an FDA designation for drugs targeting rare conditions, has been awarded to Astellas for being the first to secure approval for IA and IM treatment in patients as young as one with a triazole antifungal. This exclusivity ensures seven years of exclusive marketing rights for CRESEMBA in these indications from the date of the supplemental New Drug Application approval on Dec. 8, 2023. Pediatric exclusivity extends this period by an additional six months. Astellas had previously received orphan drug exclusivity for CRESEMBA in 2015 for IA and IM treatment in adults.

Dr. Laura Kovanda, Executive Director and Asset Lead of BioPharma Development at Astellas, expressed pride in the team's efforts in advancing research, development, and commercialization to provide CRESEMBA to pediatric patients facing limited treatment options for rare and life-threatening IA and IM infections.

Notably, CRESEMBA stands out as the sole azole antifungal therapy FDA-approved for treating IA and IM in patients as young as one.

Invasive aspergillosis and invasive mucormycosis are severe fungal infections primarily affecting immunocompromised individuals, such as those with leukemia, and can pose significant risks to pediatric patients, including morbidity and mortality.

CRESEMBA® (isavuconazonium sulfate) is indicated for treating invasive aspergillosis and invasive mucormycosis in the following forms:

  • CRESEMBA for injection: approved for adults and pediatric patients aged 1 year and older
  • CRESEMBA capsules: approved for adults and pediatric patients aged 6 years and older, weighing 16 kg and above

Before initiating antifungal therapy, it's crucial to obtain specimens for fungal culture and relevant laboratory studies for isolating and identifying the causative organism(s). Therapy may commence before the results are available, but adjustments should be made based on the findings.

Safety information for CRESEMBA highlights contraindications, warnings, and precautions. Notably, hepatic adverse drug reactions, infusion-related reactions, hypersensitivity reactions, and embryo-fetal toxicity are among the concerns. Additionally, caution is advised regarding drug interactions and the formation of drug particulates post-dilution for the intravenous formulation.

In both adult and pediatric patients, adverse reactions reported include gastrointestinal symptoms, elevated liver chemistry tests, rash, and headache, among others. Permanent discontinuation of CRESEMBA therapy during clinical trials was primarily due to adverse events such as confusional state, acute renal failure, and respiratory issues.

Overall, Astellas' announcement represents a significant advancement in addressing critical unmet medical needs for pediatric patients battling rare and life-threatening fungal infections.



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