Servier Pharmaceuticals, LLC

200 Pier Four Boulevard Boston, Massachusetts United States of America

Compounds:

Vorasidenib
Vorasidenib

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

Detailed Description: This is a signle-patient IND expanded access program (EAP) is designated 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 treatment, vorasidenib is approved and available by prescription, or the study is terminated.

Request 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.

Requesting Expanded Access

All requests for Expanded Access must be made by an appropriately licensed treating physician on behalf of the patient. Servier, will in its sole discretion, evaluate requests for EA. Servier may consider, without limitation, some of all of the following criteria:

Inclusion Criteria

Age 12 or older and weighing at least 40 Kg.

Have Grade 2 or 3 oligodendroglioma or astrocytoma per WHO 2016 or 2021 criteria. Patients with Grade 4 astrocytomas may be considered on a case-by-case basis.

Having confirmed IDH1 or IDH2 gene mutation confirmed by tissue-based diagnosis.

Have at least 1 prior surgery for glioma (biopsy, sub-total resection, gross-total resection).

Have received chemotherapy and/or radiotherapy.

Have disease recurrence or progression. Patients with stable residual disease after standard of care therapy who, in the opinion of the treating physician, are likely to gain benefit from treatment may be considered on a case-by-case basis.

Have adequate bone marrow function.

Have adequate renal function.

Have adequate cardiac function.

Exclusion Criteria

Patient is eligible for ivosidenib Patient Assistance Program.

Patient is eligible for a clinical trial with vorasidenib or ivosidenib.

Prior treatment with IDH inhibitor, unless there is isoform switching confirmed by tissue-based diagnosis.

Have a heart-rate corrected QT interval using Fridericia's formula (QTcF) >/ 450msec 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 treating physician.

Are pregnant or breastfeeding.

A treating physician may submit questions or requests for EA by emailing vorasidenib.eap@earlyaccesscare.com

Patients, Caregivers and Patient Advocates: Requests for Expanded Access must be made by a qualified physician. Servier encourages patients and caregivers to have a candid conversation about reasons for considering an investigational medicine. Your physician will be required to supervise the treatment and comply with safety reporting responsibilities. Your physician must make the written request on your behalf.

Physicians and Healthcare Professionals: Only appropriately licensed physicians may make a written request on behalf of a patient. The physician must be qualified to administer and oversee the investigational treatment and comply with Servier safety reporting responsibilities. Investigational Review Board (IRB) approval for the use of the investigational medication is required. The physician must be willing to meet and follow all applicable legal and regulatory requirements. The physician will be required to provide adequate medical history and treatment information for a specific patient, obtain informed consent, and may be required to provide additional data on the progress and safety throughout the expanded access treatment interval.

Available in These Countries:

United States of America

Vorasidenib Expanded Access Program

The vorasidenib EAP 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 continuation of 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.

Key Inclusion Criteria

Age ≥ 12 years old and weighing at least 40 kg
Grade 2 or 3 oligodendroglioma or astrocytoma per WHO 2016 or 2021 criteria. Patients with Grade 4 astrocytomas will be considered on a case-by-case basis.
Confirmed IDH1 or IDH2 gene mutation confirmed by tissue-based diagnosis
At least 1 prior surgery for glioma (biopsy, sub-total resection, gross-total resection)
Patient has previously received chemotherapy and/or radiotherapy
Have disease recurrence or progression. Patients with stable residual disease after standard of care therapy who, in the opinion of the investigator, are likely to gain benefit from treatment will be considered on a case-by-case basis.
Adequate bone marrow function.
Adequate hepatic function.
Adequate renal function.
Adequate cardiac function.

Key Exclusion Criteria:

Prior treatment with an IDH inhibitor, unless there is isoform switching confirmed by tissue-based diagnosis.
Eligible for ivosidenib Patient Assistance Program.
Eligible for a clinical trial with vorasidenib or ivosidenib.
Have a heart rate corrected QT interval using Fridericia's formula (QTcF) >450 msec or other factors that increase the risk of QT prolongation or arrythmic 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.
Are pregnant or breastfeeding.