A Regulatory Review Approach for Evaluation of Micafungin for Treatment of Neonatal Candidiasis. (18th January 2021)

• Record Type: Journal Article
• Authors: Taormina, Gillian; Gopinath, Ramya; Moore, Jason; Yasinskaya, Yuliya; Colangelo, Philip; Reynolds, Kellie; Nambiar, Sumati
• Is Part Of: Clinical infectious diseases. Volume 73:Number 12(2021)
• Page Start: 2335
• Page End: 2340
• Publication Date: 2021-01-18
• Subjects: neonatal candidiasis -- meningoencephalitis -- animal model -- regulatory approach; Communicable diseases -- Periodicals; 616.905
• DOI: 10.1093/cid/ciab025
• Languages: English
• ISSNs: 1058-4838

Abstract

Abstract: Pathogenesis of neonatal candidiasis (NC) is distinct from systemic candidiasis in adults and older pediatric patients due to the significant incidence of central nervous system involvement in neonates. Thus, although adequate and well-controlled trials in NC are often unfeasible due to difficulty enrolling patients, extrapolation of efficacy from antifungal drug trials in adults is generally not appropriate. However, treatment of NC is an area of great unmet need. We describe a regulatory review approach that combined the assessment of limited clinical efficacy, pharmacokinetics, and safety data from neonates and young infants along with microbiology outcomes and pharmacokinetic data from relevant nonclinical models of candidemia/invasive candidiasis to inform the use of micafungin in pediatric patients younger than 4 months, while communicating areas of remaining uncertainty in labeling. Abstract : The distinct pathophysiology of neonatal candidiasis necessitated an innovative regulatory pathway for evaluation of micafungin that combined assessment of limited clinical data, along with pharmacodynamic and pharmacokinetic data from relevant nonclinical models of invasive candidiasis.