Orphan drug designation for cryptococcosis medicineApril 9, 2019
Orphan drug designation has been granted to a lead drug candidate, APX001 for the treatment of cryptococcosis, said Amplyx.
APX001 had previously received orphan drug designation for the treatment of invasive candidiasis, invasive aspergillosis, coccidioidomycosis, and rare mold infections caused by Scedosporium spp., Fusarium spp., and Mucorales fungi (including Mucor spp., and Rhizopus spp.).
APX001A, the active moiety of APX001, inhibits the highly conserved fungal enzyme Gwt1, compromising growth of major fungal pathogens.
APX001 is currently in phase 2 clinical trial evaluating the efficacy and safety of both IV and oral APX001 for the first-line treatment of patients with fungal infections
Cryptococcosis is an infectious disease of the lungs or central nervous system caused by the fungus Cryptococcus (either Cryptococcus neoformans or Cryptococcus gattii), which is typically found in the environment and inhaled. Brain infections due to the fungus Cryptococcus are called cryptococcal meningitis.