Research & Development
Amplyx Pharmaceuticals passes US FDA orphan drug designation for APX001 to treat cryptococcosis
12 February 2019 -

Drug discovery company Amplyx Pharmaceuticals reported on Monday the receipt of the orphan drug designation for the lead drug candidate APX001 for treatment of cryptococcosis from the US Food and Drug Administration (FDA) Office of Orphan Product Development.

Cryptococcosis is reportedly 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. Infection is most often seen in people with a weakened immune system.

The company added that APX001 is now provided with seven years of market exclusivity in the US upon the US FDA approval of a new drug application (NDA) for the orphan designated indications. Cryptococcal Meningitis is included in the US FDA's tropical disease priority review voucher programme.

In addition to orphan designations, the US FDA had previously granted Qualified Infectious Disease Product (QIDP) designation for the company's APX001 for treatment of cryptococcosis, invasive candidiasis, invasive aspergillosis and coccidioidomycosis. QIDP provides significant development incentives including eligibility for Fast Track designation, priority review and when combined with orphan drug designation, a total of 12 years of marketing exclusivity.

According to the company, APX001A, the active moiety of APX001, inhibits 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 moulds including Fusarium spp., Scedosporium spp., and fungi from the Mucorales order.



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