The Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) for olorofim for the treatment of invasive fungal infections in patients who have limited or no treatment options.
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 is supported by data from the open-label, single-arm, phase 2b FORMULA-OLS study (ClinicalTrials.gov Identifier: NCT03583164). The trial included 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.
Data from the first 100 patients was included in the analysis. Findings showed 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).
Olorofim treatment was generally well tolerated; diarrhea, nausea, and vomiting were observed. Additionally, 8 patients experienced drug-induced liver injury that was possibly related to olorofim; 2 patients required discontinuation of treatment.
“We are committed to addressing rare fungal infections, and the acceptance of filing of olorofim NDA for use in this well-defined and high-need population marks a major milestone toward our goal of bringing new options to these patients,” said Francesco Maria Lavino, chief executive officer of F2G. “We are building an experienced commercial team in preparation for US launch, pending FDA approval. If approved, olorofim will be the first of a new class of antifungal drugs.”
A Prescription Drug User Fee Act target action date of June 17, 2023 has been set for the application.
- F2G announces FDA filing acceptance of New Drug Application for olorofim for the treatment of invasive fungal infections. News release. F2G Inc. Accessed December 19, 2022. https://www.globenewswire.com/news-release/2022/12/19/2575954/0/en/F2G-Announces-FDA-Filing-Acceptance-of-New-Drug-Application-for-Olorofim-for-the-Treatment-of-Invasive-Fungal-Infections.html.
- F2G announces data from phase 2b study at ID Week 2022 showing positive therapeutic response in patients with invasive fungal infections treated with olorofim. News release. F2G Inc. October 21, 2022. Accessed December 19, 2022. https://www.globenewswire.com/news-release/2022/10/21/2539410/0/en/F2G-Announces-Data-from-Phase-2b-Study-at-ID-Week-2022-Showing-Positive-Therapeutic-Response-in-Patients-with-Invasive-Fungal-Infections-Treated-with-Olorofim.html.