New Potential Treatment for Cryptococcosis Granted Orphan Drug Designation


Cryptococcosis is a disease caused by the Cryptococcus fungus. It can affect the lungs, the spinal cord, or the brain. When the brain is infected, the condition is called cryptococcal meningitis.

Unfortunately, cryptococcosis is just one of many fungal infections that has a high unmet need. For instance, infections caused by Mucorales, Fusarium, Scedosporium, and Aspergillus fungi have a mortality rate between 50-80%. The standard treatment for fungal infections is amphotecricin B and flucytosine, however it is often ineffective. Additionally, this therapy is known to cause serious side effects (such as kidney toxicity and anemia). It requires hospitalization which affects patient’s quality of life, and more fungi is becoming resistant to it as time goes on.

Most patients who develop cryptococcosis already have a weak immune system from HIV/AIDS, organ transplant, high doses of corticosteroids, Hodgkin’s disease, or cancer. The standard therapy has extended life for some patients. However, for HIV patients in particular, cryptococcal meningitis is still the leading cause of mortality. This is especially true in lower-income countries.

To address this clear need, Amplyx Pharmaceuticals has been diligently investigating APX001 as a potential treatment for cryptococcosis. 

Development of APX001

APX001 has just received Orphan Drug Designation by the FDA for the treatment of cryptococcosis. Previously, it had received this designation as well as Qualified Infectious Disease Product Designation for coccidioidomycosis, aspergillosis, candidiasis, and other mold infections caused be either Mucorales fungi, Fusarium spp., or Scedosporium spp.

Researchers believe that when used in combination with fluconazole, this therapy could be an effective, transformational therapy for people with cryptococcosis, specifically aiding in the treatment of cryptococcal meningitis. Amplyx is developing the drug as an oral treatment that is taken once a day.

You can read more about this new potential therapy for cryptococcosis here.

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