Vorasidenib Expanded Access Program

This is an expanded access program to provide vorasidenib for treatment of patients 12 years or older with IDH1- or IDH2-mutated glioma.

This expanded access program is designed to provide access to vorasidenib for patients with IDH1- or IDH2-mutated glioma who are not eligible for other vorasidenib clinical trials, and who in the opinion of the treating oncologist would potentially benefit from treatment with vorasidenib. Safety assessments (including vital signs, hematology, and serum chemistry) occur every two weeks for the first two cycles (28 day each cycle), then monthly for the duration of treatment. Treatment with vorasidenib will continue until, in the clinical judgement of the treating physician, the patient is no longer benefiting from the treatment, vorasidenib is approved and available by prescription, or the study is terminated. Requests by treating physicians to file a single patient investigational new drug application as part of the expanded access program for vorasidenib will be considered on a case-by-case basis.

Glioma, Recurrence, Disease Attributes, Pathologic Processes, Neoplasms, Neuroepithelial, Neuroectodermal Tumors, Neoplasms, Germ Cell and Embryonal, Neoplasms by Histologic Type, Neoplasms, Neoplasms, Glandular and Epithelial, Neoplasms, Nerve Tissue

Inclusion Criteria: Age ≥ 12 years old and weighing at least 40 kg. Have IDH-mutant oligodendroglioma or astrocytoma per WHO 2021 criteria, with the IDH1 or IDH2 gene mutation confirmed by tissue-based diagnosis. Have at least 1 prior surgery for glioma (including biopsy). Is not in immediate need of chemotherapy and/or radiotherapy based on the clinical judgement of the treating oncologist. Have adequate bone marrow function. Have adequate hepatic function. Have adequate renal function. Have adequate cardiac function. Exclusion Criteria: Patient has IDH1-mutant glioma that is predominantly contrast-enhancing and the patient is eligible for ivosidenib Patient Assistance Program or able to access ivosidenib through a third-party payer. Patients whose disease progresses after treatment with ivosidenib or who are unable to tolerate ivosidenib may be eligible Patient is eligible for a clinical trial with vorasidenib. (Note that patients who are enrolled in a Servier-sponsored clinical trial and have completed all requirements of the trial may be eligible if the patient continues to benefit from vorasidenib and does not meet criteria for discontinuation of treatment) Patient has a grade 4 tumor and has not received appropriate standard of care or been approved for an exception by a Servier-designated panel of independent experts. Patient has a heart-rate corrected QT interval using Fridericia's formula (QTcF) ≥450 msec or other factors that increase the risk of QT prolongation or arrhythmic events (e.g., heart failure, hypokalemia, family history of long QT interval syndrome). Subjects with bundle branch block and prolonged QTcF may be eligible at the discretion of Servier Pharmaceuticals and the investigator. Patient is pregnant or breastfeeding.