FDA Grants Orphan Drug Designation for Cryptococcosis Treatment

The US Food and Drug Administration (FDA) has granted orphan drug designation to APX001 for the treatment of cryptococcosis.

APX001, a leading candidate developed by Amplyx Pharmaceuticals, is an antifungal that 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). The FDA also previously granted Qualified Infectious Disease Product designation for APX001 for treatment of cryptococcosis, invasive candidiasis, invasive aspergillosis, and coccidioidomycosis, according to an Amplyx statement.

Cryptococcosis is caused by the fungus Cryptococcus and infects the lungs and central nervous system. Brain infections with Cryptococcus are called cryptococcal meningitis and most commonly infect people with weakened or suppressed immune systems, such as those with HIV/AIDS, Hodgkin's disease, or who have had an organ transplant.

"APX001 is a really exciting new potential treatment for serious life-threatening fungal infections," Ciara Kennedy, PhD, MBA, president and CEO of Amplyx Pharmaceuticals, told Contagion^® in October 2017, when phase 1 data were released on APX001. "It is a small molecule inhibitor of an enzyme called GWT1 that's within the fungal cell, and it is very broad spectrum of activity because GWT1 is highly conserved across fungal species so we have now a tremendous amount of non-clinical data to support its activity."

The orphan drug designation allows APX001 7 years of market exclusivity in the US, and QIDP provides eligibility for Fast Track designation, priority review, and a combined 12 years of marketing exclusivity when combined with orphan drug designation.

"Orphan and QIDP designations highlight the potential for APX001 to address unmet needs of patients with rare, life-threatening infections, and with the FDA's recent addition of cryptococcal meningitis to its list of neglected tropical diseases, Amplyx has the potential to obtain a valuable Tropical Disease Priority Review Voucher from the FDA, creating additional value for Amplyx and its stakeholders," Dr. Kennedy said in a statement.