FDA Needs More Data Before Deciding on Novel Antifungal Olorofim
The Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) regarding the New Drug Application (NDA) for olorofim for the treatment of invasive fungal infections in patients who have limited or no treatment options.
According to the FDA letter, additional data will be needed before a decision on approval can be made. Olorofim is part of a new class of antifungal agents called orotomides, which work by targeting a key enzyme in the de novo pyrimidine biosynthesis pathway.
The NDA included data from the open-label, single-arm, phase 2b FORMULA-OLS study (ClinicalTrials.gov Identifier: NCT03583164). The trial enrolled patients who had limited or no treatment options for either proven invasive fungal disease (including aspergillosis, lomentosporiosis, scedosporiosis, Scopulariopsis infections, and refractory extrapulmonary coccidioidomycosis) or probable pulmonary invasive aspergillosis.
Findings based on an analysis of the first 100 study participants showed that 44% of patients had a complete or partial response to treatment at day 42 (primary endpoint). All-cause mortality was reported to be 15% and 20% at day 42 and day 84, respectively (secondary endpoint).
“While F2G is disappointed with this outcome, we remain optimistic about olorofim’s potential to address an unmet need for patients with invasive fungal infections who have exhausted their treatment alternatives,” said Francesco Maria Lavino, CEO of F2G. “We are assessing the details of the CRL, and we plan to meet with the FDA to discuss it further, but we are confident that we can identify a regulatory path forward in the US.”
