FDA Awards Orphan Drug Designation to FG001 in High-Grade Glioma

The FDA has granted an orphan drug designation to FG001 as an optical imaging agent for the visualization of malignant tissue during surgery for patients with high-grade glioma, according to an announcement from FluoGuide.¹

FG001 is a fluorophore designed to target uPAR, a cancer-specific target expressed in a variety of solid tumors. Because FG001 has the same spectral specifications as the previously approved indocyanine green, the agent could be integrated into use with existing imaging equipment without the need for modifications.

The optical imaging agent is administered intravenously prior to surgery, where FG001 then illuminates cancerous tissue, providing guidance to surgeons in the precise removal of cancer while preserving healthy tissue. Notably, FG001 is being evaluated in a controlled, randomized, multicenter phase 2b trial (FG001-CT-001).

“The granting of orphan designation by the FDA is important and may simplify FG001's path toward market approval and can provide significant benefits in conducting clinical trials, as well as during commercialization,” Morten Albrechtsen, chief executive officer of FluoGuide, stated in a news release. “We have completed enrollment and treatment in our phase llb clinical trial of FG001 and are now looking forward to top line results, which we expect by the end of November.”

In June 2023, FluoGuide announced positive interim results from phase 2a trial of FG001 in patients with head and neck cancer, where FG001 successfully lit tumors in 100% of the patients.²

The phase 2b trial being conducted out of Copenhagen, Denmark, is enrolling patients at least 18 years of age with primary malignant glioblastoma scheduled for neurosurgery, and it will evaluate the effectiveness of FG001 in guiding the surgery of patients compared with 5-ALA and white light.³ Patients must not previously have received FG001, and they are included only if the surgery is planned to be guided by 5-ALA. Anticipated gross total resection is required, along with adequate organ and bone marrow function.

Exclusion criteria include those with any known allergy or hypersensitivity to indocyanine green, have a performance status or co-morbidity deeming a patient unfit for participation in the trial, and pre-existing hepatic and/or renal insufficiency.

Patients will be randomly assigned in a 1:1 fashion between FG001 and 5-ALA, comparing FG001 and 5-ALA to white light in their respective arm. Notably, the trial is not powered to demonstrate statistical significance between FG001, white light or 5-ALA.¹

The primary end points of the phase 2 portion of the trial include pharmacokinetics of FG001; evaluating the efficacy of the agent following surgery by determination of gross total resection; positive predicted value of FG001 and 5-ALA fluorescence tumor tissue obtained from each trial patient; contrast of FG001 to differentiate tumor tissue from normal tissue; negative predictive value from each trial patient for FG001 vs 5-ALA; safety and tolerability of the optimal dose of FG001.³

“We are now discussing plans for next stage clinical development of FG001 in aggressive brain cancer with regulators, so we can ensure the most effective route to approval, supported by the necessary data," Albrechtsen added.¹

Topline results of the phase 2b trial are expected to be read out before the end of November 2023.

References

1. FluoGuide receives FDA orphan drug designation for FG001 in high-grade glioma. News Release. FluoGuide. October 3, 2023. Accessed October 3, 2023. https://fluoguide.com/mfn_news/fluoguide-receives-fda-orphan-drug-designation-for-fg001-in-high-grade-glioma/
2. FluoGuide announces positive interim results from phase IIa trial of FG001 in head & neck cancer. Modular Finance. June 20, 2023. Accessed October 3, 2023. https://fluoguide.com/mfn_news/fluoguide-announces-positive-interim-results-from-phase-iia-trial-of-fg001-in-head-neck-cancer/
3. An open-label, dose escalation, single-dose administration, multi-center phase I/II trial of FG001 (an imaging agent), in patients with glioblastoma scheduled for neurosurgery. ClinicalTrialsRegister.eu. June 10, 2021. Accessed October 3, 2023. https://www.clinicaltrialsregister.eu/ctr-search/trial/2020-003089-38/SE