Exagamglogene Autotemcel Dosage

Medically reviewed by Drugs.com. Last updated on Jan 30, 2024.

Usual Adult Dose for Sickle Cell Disease

MINIMUM RECOMMENDED DOSE:
3 x 10(6) CD34+ cells/kg administered as a single-dose IV infusion between 48 hours and 7 days after the last dose of full myeloablative conditioning.

MOBILIZATION AND APHERESIS:

• Perform prior to conditioning and treatment to isolate CD34+ cells for drug manufacturing.
• Total collection target: At least 20 x 10(6) CD34+ cells/kg; perform 2 consecutive days of cell collection per cycle if clinically tolerated
• Back-up unmodified rescue cells: At least 2 x 10(6) CD34+ cells/kg required; can be collected on the 3rd day of collection

MYELOABLATIVE CONDITIONING:

• Initiate conditioning only after the total dose of this drug (plus rescue cells) are received.
• Administer full myeloablative conditioning before starting treatment with this drug.
• Blood transfusion is recommended at least 8 weeks prior to initiation of myeloablative conditioning, to maintain hemoglobin S (HbS) levels of less than 30% of total hemoglobin (Hb) and keep total Hb concentration at 11 g/dL or less.

Comments:

• See the Lot Information Sheet provided with the product shipment for additional information about the number of vials required to achieve the patient-specific dose. Administer all vials.
• Refer to prescribing information for the mobilization and myeloablative conditioning agents prior to treatment.
• Prior to apheresis, transfusion is recommended to maintain HbS levels of less than 30% of total Hb and keep total Hb concentration at 11 g/dL or less.
• If the minimum dose is not met after initial product manufacturing, the patient should undergo additional cycles of mobilization and apheresis; each cycle must be separated by a minimum of 14 days.
• The collected 2 x 10(6) CD34+ cells/kg or more of unmodified backup cells should be cryopreserved before myeloablative conditioning and infusion with this drug.
• Administer an antipyretic (e.g., acetaminophen) and an antihistamine (e.g., diphenhydramine hydrochloride) prior to administering this drug.

Use: For the treatment of sickle cell disease in patients with recurrent vaso-occlusive crises

Usual Pediatric Dose for Sickle Cell Disease

12 to less than 18 years:
MINIMUM RECOMMENDED DOSE:
3 x 10(6) CD34+ cells/kg IV administered as a single dose

MOBILIZATION AND APHERESIS:

• Total collection target: At least 20 x 10(6) CD34+ cells/kg; perform 2 consecutive days of cell collection per cycle if clinically tolerated
• Back-up unmodified rescue cells: At least 2 x 10(6) CD34+ cells/kg required; can be collected on the 3rd day of collection

MYELOABLATIVE CONDITIONING:

• This drug must be administered between 48 hours and 7 days after the last dose of full myeloablative conditioning.
• Blood transfusion is recommended at least 8 weeks prior to initiation of myeloablative conditioning, to maintain hemoglobin S (HbS) levels of less than 30% of total hemoglobin (Hb) and keep total Hb concentration at 11 g/dL or less.

Comments:

• See the Lot Information Sheet provided with the product shipment for additional information about the number of vials required to achieve the patient-specific dose. Administer all vials.
• Refer to prescribing information for the mobilization and myeloablative conditioning agents prior to treatment.
• Prior to apheresis, transfusion is recommended to maintain HbS levels of less than 30% of total Hb and keep total Hb concentration at 11 g/dL or less.
• If the minimum dose is not met after initial product manufacturing, the patient should undergo additional cycles of mobilization and apheresis; each cycle must be separated by a minimum of 14 days.
• The collected 2 x 10(6) CD34+ cells/kg or more of unmodified backup cells should be cryopreserved before myeloablative conditioning and infusion with this drug.
• Administer an antipyretic (e.g., acetaminophen) and an antihistamine (e.g., diphenhydramine hydrochloride) before administering this drug.

Use:
For the treatment of sickle cell disease in patients with recurrent vaso-occlusive crises

Renal Dose Adjustments

Renal Dysfunction (estimated glomerular filtration rate less than 60 mL/min/1.73 m2): This drug has not been studied in this patient population

Comment:

• Patients should be assessed for renal impairment to ensure that hematopoietic stem cell (HSC) transplantation is appropriate.

Liver Dose Adjustments

Liver Dysfunction: This drug has not been studied in this patient population

Comment:

• Patients should be assessed for hepatic impairment to ensure that HSC transplantation is appropriate.

Precautions

CONTRAINDICATIONS: None

Safety and efficacy have not been established in patients younger than 12 years.

Consult WARNINGS section for additional precautions.

Dialysis

Data not available

Other Comments

Administration advice:

• For autologous, one-time, single-dose, IV use only.
• This drug should not be administered to patients with active HIV-1, HIV-2, HBV, or HCV.
• Verify that the patient's identity matches the unique patient identification information on the product labels and lot information sheet prior to thaw and infusion.
• Irradiate blood products required within the first 3 months after treatment with this drug.
• Patients treated with this drug should not donate, organs, tissues, or cells at any time in the future.
• The manufacturer product information should be consulted for detailed instructions prior to the administration of this drug.

Mobilization and Apheresis:

• Confirm that hematopoietic stem cell (HSC) transplantation is appropriate for the patient before initiating mobilization, apheresis, and myeloablative conditioning.
• Granulocyte-colony stimulating factor should not be used for mobilization in sickle cell disease patients.
• Discontinue disease-modifying therapies for sickle cell disease (e.g., hydroxyurea, crizanlizumab, voxelotor) 8 weeks before the planned start of mobilization and conditioning.
• The unmodified backup cells may be needed for rescue treatment under one of the following conditions:
• compromise of this drug after initiation of myeloablative conditioning and before infusion of this drug
• neutrophil engraftment failure; or
• loss of engraftment after infusion with this drug

Myeloablative Conditioning:

• Myeloablative conditioning should not be initiated until the complete set of vial(s) comprising the total dose of this drug has been received and stored at the treatment center and the availability of the backup collection of unmodified rescue cells is confirmed.
• Discontinue iron chelation therapy at least 7 days before myeloablative conditioning. Iron chelation may be restarted after administration of this drug based on clinical practice.
• Avoid the use of non-myelosuppressive iron chelators for at least 3 months and use of myelosuppressive iron chelators for at least 6 months after treatment with this drug. Phlebotomy is an alternative to iron chelation when appropriate.
• Seizure prophylaxis should be considered prior to initiating myeloablative conditioning.

Storage requirements:

• Vials of this drug should be stored in the vapor phase of liquid nitrogen at -135C or less (-211F or less) until ready for thaw and administration.
• Administer one vial at a time within 20 minutes of thawing.
• Do not re-freeze this drug after thawing.

Reconstitution/preparation techniques:

• The manufacturer product information should be consulted.

General:

• Contact Vertex Pharmaceuticals Incorporated at 1-877-634-8789 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch to report suspected adverse reactions.

Monitoring:

• Hepatic: HBV and HCV (before initiating therapy)
• Hematologic: Absolute neutrophil count and platelets (periodically)
• Infection: HIV-1, HIV-2 (before initiation therapy)
• Reproductive: Serum pregnancy (before start of each mobilization cycle and before myeloablative conditioning)

Patient advice:

• Read the US FDA-approved patient labeling (Patient Information).
• There is an increased risk for bleeding from initiation of myeloablative conditioning until platelet engraftment is achieved.
• Do not donate blood, organs, tissues, or cells at any time in the future.
• Contact your healthcare provider immediately if you have:
• symptoms such as fever, chills, severe headache, abnormal bruising, prolonged bleeding
• nosebleeds, bleeding gums, blood in your urine, stool, or vomit, or coughing up blood
• Inform your healthcare provider of all medications, herbal and dietary supplements.
• Women are advised not to breastfeed during myeloablative conditioning due to the potential for serious adverse effects.
• Women of childbearing potential and males of reproductive potential must use effective contraception from the start of mobilization through at least 6 months after administration of this drug.

See also:

Further information

Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.

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