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# Orphan Designation for APX001 Treating Cryptococcosis
- URL: https://www.fdaweb.com/orphan-designation-for-apx001-treating-cryptococcosis/
- Published: 2019-02-11T12:00:00.000Z
- Updated: 2026-09-15T01:02:52.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5143351

FDA has granted Amplyx Pharmaceuticals an orphan drug designation for APX001 and its use for treating cryptococcosis. The therapy has previously received an orphan drug designation for treating invasive candidiasis, invasive aspergillosis, coccidioidomycosis, and rare mold infections caused by *Scedosporium* spp., *Fusarium* spp., and Mucorales fungi (including *Mucor* spp., and *Rhizopus* spp.).

Cryptococcosis is an infectious disease of the lungs or central nervous system (the brain or spinal cord) caused by the fungus *Cryptococcus* (either *Cryptococcus neoformans* or *Cryptococcus gattii),* which is typically found in the environment and inhaled. Brain infections caused by the fungus are called cryptococcal meningitis. Infection is most often seen in people with a weakened immune system, including those who are infected with HIV/AIDS, take high doses of corticosteroid medicines, have had an organ transplant, are receiving immune suppressing therapies for cancer or other diseases, or have Hodgkin's disease, the company says.

Amplyx says APX001A works by inhibiting the highly conserved fungal enzyme Gwt1, compromising growth of major fungal pathogens. “In multiple nonclinical studies, APX001 has shown broad-spectrum activity against common species of *Candida* spp. and *Aspergillus* spp., including multi-drug resistant strains including *Candida auris* and rare, hard-to-treat molds including *Fusarium* spp., *Scedosporium* spp., and fungi from the Mucorales order,” it says.