Gallium maltolate now has been granted an orphan drug designation for adult and pediatric patients with glioblastoma multiforme.
The FDA has granted an orphan drug designation (ODD) for gallium maltolate (GaM) for the treatment of pediatric patients with glioblastoma multiforme (GBM), according to Imaging Biometrics, LLC.1
"We are very pleased that the FDA has now confirmed that the orphan drug designation for GaM applies to all GBM populations including pediatric patients," said Trevor Brown, chief executive officer of IQ-AI, in a press release.
Previously in February 2023, the agent was granted an orphan drug designation by the FDA for adult patients with GBM. Since then, the FDA has confirmed that the designation also applies to pediatric patients.
In a preclinical study of oral GaM in pediatric patients with GBM, a significant survival benefit was observed, including a median overall survival with a duration that more than doubled compared with the untreated controls.
Coupled with the easy administration by capsule, these findings show the potential benefit of GaM for both pediatric and adult patients.
A prospective, single-center, single-arm, open-label, phase 1 study (NCT04319276) is currently evaluating the the preliminary efficacy and safety agent in patients with relapsed/refractory GBM.2 The trial plans to enroll 24 patients into the dose-escalation part and will follow a 3 + 3 phase 1 dose-escalation design where they will be treated with GaM at doses of 500 mg every other day, 1,000 md daily, or 1,500 mg daily.
Enrollment in the study is open to patients aged 18 years and older with a prior histological diagnosis of GBM who have received standard treatment with radiotherapy and temozolomide (Temodal). Patients must have an ECOG performance status of 0-2, measurable disease, adequate bone marrow function, and adequate hepatic and renal function. If patients are taking oral iron supplements or iron chelators, they must be discontinued at least 1 week prior to starting GaM as they may impact on the efficacy of GaM.
Female patients are required to be either postmenopausal for at least 1 year prior to enrollment, be surgically sterile, agree to practice two acceptable methods of contraception , or agree to practice abstinence. Even if surgically sterile, male patients must agree to either practice effective barrier contraception during the entire study period and through 60 calendar days after the last dose of study agent, or practice true abstinence.
The study will exclude patients with the presence of other active malignant disease diagnosed within 12 months, those who have received prior chemotherapy or radiotherapy within 14 days of study entry, and those with known hypersensitivity to or intolerance to gallium-based medications. Patients may not have concurrent use of cytotoxic chemotherapy, unstable or severe concurrent medical conditions, and a history of interstitial lung disease, history of slowly progressive dyspnea and unproductive cough, sarcoidosis, silicosis, idiopathic pulmonary fibrosis, pulmonary hypersensitivity pneumonitis, or symptomatic pleural effusion.
The primary end point of the study is assessing the maximum-tolerated dose and secondary end points are progression-free survival, overall survival, and dose-limiting toxicity.