Target product profile and discovery and development path for novel cryptococcal disease treatments

by Meya DB, De Rycker M, Gilbert IH, Hope W, Jelagat Odionyi J, Keegane M, Loysef A, Moral-Lopezg P, Williamson PR, Tane LK, Ribeiro I, Miles TJ. Journal of Infection 2025, 91(5):106643. doi: 10.1016/j.jinf.2025.106643

Summary: The World Health Organization has designated Cryptococcus neoformans and Cryptococcus gattii as critical and medium priority pathogens, respectively. These mainly impact people with HIV residing in low- and middle-income countries, but other patient groups and settings are also affected. The authors of this review discuss the global burden of cryptococcal disease, the limitations of currently available treatments, the development of a target product profile (TPP) for much-needed novel cryptococcal disease treatments, the minimal and preferred attributes of these novel treatments, and potential drug discovery and development pathways. For the TPP, key attributes are tolerability, activity across disease subtypes, and regimen simplicity. Other attributes include widespread suitability and manageable drug-drug interactions.

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