The Food and Drug Administration (FDA) has granted Orphan Drug designation to APX001 (Amplyx Pharmaceuticals) for the treatment of cryptococcosis.

Cryptococcosis is a fungal disease that affects the lungs or central nervous system and is caused by either Cryptococcus neoformans or Cryptococcus gattii, 2 species of the fungus Cryptococcus which are typically found in the environment. Individuals become infected after breathing in the microscopic fungus; those with compromised immune systems (HIV/AIDS) are most often affected.

APX001 is a broad-spectrum antifungal that works by inhibiting the fungal enzyme Gwt1. A Phase 2 trial – for both an IV and oral formulation – is currently underway; the trial is evaluating the safety and efficacy of APX001 as a first-line treatment in patients with fungal infections.

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“While antiretroviral therapy has successfully extended the lifespan of HIV patients, cryptococcal meningitis remains a leading cause of death in HIV patients, particularly in low and middle-income countries,” said Michael Hodges, MD, Chief Medical Officer of Amplyx. “The standard therapy of intravenous amphotericin B plus flucytosine requires inpatient hospitalization and has been known to cause significant side effects including anemia and kidney toxicity. APX001, Amplyx’s first-in-class antifungal agent, in combination with fluconazole, has the potential to be a transformational life-saving, once daily, all oral treatment for cryptococcal meningitis.”

The FDA has already granted APX001 Qualified Infectious Disease Product (QIDP) designation for the treatment of cryptococcosis, as well as for invasive candidiasis, invasive aspergillosis, and coccidioidomycosis.

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