Why is this medication prescribed?

Exagamglogene autotemcel is prescribed for sickle cell disease (SCD), an inherited disorder of the red blood cells that causes disruption of blood flow, in adults and children 12 years of age and older, with a history of vaso-occlusive events (complications from SCD that can include pain crises, chest pain, and stroke), or transfusion-dependent beta thalassemia (TDT), an inherited blood disorder in which the body does not produce enough beta globin, which is needed to make hemoglobin, necessitating blood transfusions in order to survive. Within the class of drugs known as autologous cellular immunotherapy, which is a form of medicine made from the patient’s own blood cells, exagamglogene autotemcel is found. It functions by assisting the body in producing hemoglobin, which in TDT patients helps manufacture more healthy hemoglobin and helps prevent sickling of cells in SCD patients.

How should this medicine be used?

In a hospital or infusion facility, a physician or nurse will inject exagamglogene autotemcel intravenously (into a vein) as a suspension (liquid). Your doctor or nurse will give you other chemotherapy medications to help your body get ready for exagamglogene autotemcel before your exagamglogene autotemcel dose.

A blood stem cell sample will be obtained from you in a hospital or treatment facility via a procedure known as mobilization and apheresis, which involves removing blood stem cells from the body, prior to the administration of your dosage of exagamglogene autotemcel injection. It could be necessary to repeat this process, which typically takes a week. To make an injection dose of exagamglogene autotemcel, the manufacturer will receive the sample. Your blood stem cells may not be ready to be administered to you for up to six months following their collection. This medicine should only be administered to you because it is derived from your own cells.

Once you receive your dose of exagamglogene autotemcel, you will need to stay in the hospital for 4-6 weeks while your condition is monitored for any side effects. Consult your physician about what to anticipate before, during, and after the procedure for mobilization and apheresis.

Request a copy of the patient’s manufacturer’s information from your pharmacist or physician.

Other uses for this medicine

Ask your doctor or pharmacist for more information if you think this medicine could be recommended for something else.

What special precautions should I follow?

Special Precautions for Exagamglogene autotemcel:

• If you have an allergy to any of the substances in exagamglogene autotemcel injection, dimethyl sulfoxide (DMSO), any other drugs, or exagamglogene autotemcel itself, let your doctor and pharmacist know. For an ingredient list, consult the Medication Guide or ask your pharmacist.
• Inform your physician and pharmacist about all of the vitamins, nutritional supplements, herbal items, prescription and over-the-counter drugs, and vitamins you now take or intend to take.
• Iron chelators are a list of over-the-counter or herbal medications that might interact with exagamglogene autotemcel. Before taking lovotibeglogene autotemcel, make sure your doctor and pharmacist are aware that you are taking these drugs. If you are using exagamglogene autotemcel, do not begin any new medicine without first consulting your doctor.
• Inform your physician if you currently have low platelets, liver disease, or kidney illness. Inform your physician if you have been infected with the hepatitis B virus (HBV), hepatitis C virus (HCV), or the human immunodeficiency virus (HIV). Inform your physician of any prior hematopoietic stem cell (HSC) transplants.
• Inform your physician if you intend to get pregnant or are already pregnant. Pregnancy should not occur while you are taking an injection of exagamglogene autotemcel. A pregnancy test must be completed prior to mobilization, and you should begin using birth control during the six months following the injection of exagamglogene autotemcel to prevent conception. You should take birth control to avoid getting pregnant while undergoing therapy and for six months following your injection of exagamglogene autotemcel if you are a man and your female partner is potentially pregnant. Consult your physician about birth control options that are suitable for you. Call your doctor right away if you or your partner become pregnant while having the exagamglogene autotemcel injection. Injections of exagamglogene autotemcel may be harmful to fetuses.
• Inform your physician if you are nursing a child or intend to do so. If you are receiving exagamglogene autotemcel injectable therapy, you should not breastfeed.
• Donating blood, organs, tissues, or cells for transplantation is not advised after receiving an injection of exagamglogene autotemcel.

What special dietary instructions should I follow?

There are typically no specific dietary restrictions directly related to Exagamglogene autotemcel therapy itself. However, patients with β-thalassemia may have dietary considerations related to their underlying condition, such as ensuring adequate iron intake if not on chelation therapy or maintaining a balanced diet to support overall health and well-being.

What should I do if I forget a dose?

Exagamglogene autotemcel is a gene therapy administered as a single treatment rather than as repeated doses. However, if you are referring to missed appointments or follow-up visits, it’s important to reschedule as soon as possible. Regular monitoring and follow-up are critical components of post-gene therapy care to monitor for efficacy and any potential adverse effects.

What side effects can this medication cause?

Here are some potential side effects associated with Exagamglogene autotemcel:

• Infusion-related Reactions: These may include fever, chills, nausea, vomiting, headache, or other flu-like symptoms shortly after infusion.
• Bone Marrow Suppression: Gene therapy involves a conditioning regimen which may temporarily suppress bone marrow function. This can lead to decreased production of blood cells (anemia, thrombocytopenia, neutropenia).
• Infections: Patients undergoing gene therapy are at increased risk of infections due to the immunosuppressive effects of conditioning regimens and other treatments.
• Risk of Graft-versus-Host Disease (GVHD): GVHD is a potential complication where the transplanted cells (in this case, genetically modified stem cells) recognize the recipient’s body as foreign and attack the tissues, leading to inflammation and organ damage.
• Adverse Events Related to the Viral Vector: Exagamglogene autotemcel uses a viral vector to deliver the therapeutic gene. While efforts are made to minimize risk, there can be immune responses or other reactions related to the viral vector.
• Long-term Risks: Due to the novelty of gene therapy, long-term effects are still being studied. Potential risks could include the development of leukemia or other malignancies, although these risks appear to be relatively low based on current data.
• Other Potential Complications: Depending on individual patient factors and the specifics of treatment, there may be other less common side effects or complications.

It’s important for patients undergoing Exagamglogene autotemcel therapy to be closely monitored by healthcare professionals who are experienced in managing complications associated with gene therapy and bone marrow transplantation. Patients and caregivers should be educated about potential side effects, signs of complications, and the importance of adherence to follow-up appointments for ongoing assessment and management.

What should I know about storage and disposal of this medication?

Storage and Disposal:

• Storage: Exagamglogene autotemcel is typically stored by the healthcare provider or facility that administers the treatment. It should be stored according to specific instructions provided by the manufacturer, which may include refrigeration or freezing depending on the formulation.
• Disposal: Any unused portions or materials associated with the administration of Exagamglogene autotemcel should be disposed of properly according to local regulations and healthcare facility protocols. This may involve special handling procedures due to the biological nature of the therapy.

In case of emergency/overdose

• Emergency: In case of any unexpected adverse reactions or emergencies during or after administration, it’s crucial to seek immediate medical attention. Healthcare providers administering Exagamglogene autotemcel should be prepared to manage potential complications according to established clinical protocols.
• Overdose: Due to the nature of gene therapy and the specific administration protocols involved, the concept of overdose may not apply in the traditional sense. However, if there are concerns about an excessive response or unexpected side effects, healthcare providers should assess and manage the patient accordingly.

What other information should I know?

• Monitoring: Patients receiving Exagamglogene autotemcel require thorough monitoring post-treatment to evaluate the efficacy of the gene therapy and manage any potential side effects or complications.
• Follow-Up: Long-term follow-up is essential to assess the durability of the therapeutic effect and monitor for any late-onset adverse events.
• Patient Education: Patients and caregivers should receive comprehensive education about the therapy, including its purpose, administration process, potential benefits, risks, and the importance of adhering to follow-up appointments.

Regulatory Guidance: Always follow the specific guidance provided by the prescribing healthcare provider or the manufacturer regarding storage, handling, administration, and disposal of Exagamglogene autotemcel. Local regulations and institutional policies may also provide additional directives for safe and effective use.

It’s crucial for patients and healthcare providers to maintain open communication regarding any questions or concerns about Exagamglogene autotemcel to ensure safe and effective treatment.