Frequently asked questions about treatment with LYFGENIA™
Vaso-occlusive events (VOEs) were defined as any of the following events requiring evaluation at a medical facility:
- an episode of acute pain with no medically determined cause other than vaso-occlusion, lasting more than 2 hours1
- acute chest syndrome (ACS)1
- acute hepatic sequestration1
- acute splenic sequestration1
Severe VOEs (sVOEs) were defined as either of the following events:
- VOE requiring a hospitalization or multiple visits to an emergency department/urgent care over 72 hours and receiving intravenous medications at each visit1
- priapism requiring any level of medical attention1
A hospital that has been carefully selected based on expertise in areas such as transplants or cell and gene therapy, with staff trained to administer LYFGENIA
About LYFGENIA
LYFGENIA is a one-time, single-dose, autologous gene addition therapy that enables patients to produce a form of anti-sickling adult hemoglobin (HbAT87Q). LYFGENIA adds functional copies of a modified form of the β-globin gene (βA-T87Q-globin gene) into patients’ hematopoietic stem cells (HSCs) through transduction of autologous CD34+ cells with BB305 LVV. After LYFGENIA infusion, the transduced CD34+ HSCs engraft in the bone marrow and produce red blood cells containing βA-T87Q-globin that can combine with α-globin to produce functional HbA containing βA-T87Q-globin.1
LYFGENIA is shipped from the manufacturing facility to the treatment center storage facility in a cryoshipper, which may contain multiple metal cassettes intended for a single patient. A Lot Information Sheet is affixed inside the shipper.1
LYFGENIA is supplied in 1 to 4 infusion bags containing a frozen suspension of genetically modified autologous cells, enriched for CD34+ cells. Each bag contains approximately 20 mL. Each infusion bag is individually packed within an overwrap in a metal cassette. LYFGENIA is shipped from the manufacturing facility to the treatment center storage facility in a cryoshipper, which may contain multiple metal cassettes intended for a single patient. LYFGENIA is for autologous use only, so the patient’s identity must match patient identifiers on the product. Thaw LYFGENIA in a water/dry bath and administer the product within 4 hours. Each bag is administered via intravenous infusion over a period of less than 30 minutes.1
LYFGENIA is a one-time, single-dose, autologous gene addition therapy that is administered at a Qualified Treatment Center (QTC) with 6 important key steps in the treatment process. Time frames for treatment may vary.1
No. LYFGENIA is powered by a third-generation lentiviral vector (LVV). LVVs are built using a blueprint of the HIV virus—not the actual virus itself. They do not contain the parts of the virus that can cause an infection.2-4
Patients may have a false-positive test for HIV if tested using a PCR-based test after being treated with LYFGENIA. Other tests such as antigen tests or nucleic acid tests may be able to accurately assess a person’s HIV status.1,5
Clinical Trials & FDA Approval
LYFGENIA was FDA approved for use in the US on December 8, 2023. LYFGENIA is not approved for use outside the US.1
LYFGENIA is indicated for the treatment of patients 12 years of age or older with sickle cell disease and a history of vaso-occlusive events.1
Limitations of Use: Following treatment with LYFGENIA, patients with α-thalassemia trait (-α3.7/-α3.7) may experience anemia with erythroid dysplasia that may require chronic red blood cell transfusions. LYFGENIA has not been studied in patients with more than two α-globin gene deletions.1
LYFGENIA is indicated for the treatment of patients 12 years of age or older with sickle cell disease and a history of vaso-occlusive events. Patients with a history of stroke or vasculopathy who fit the indication criteria are still eligible to receive LYFGENIA at the recommendation of their healthcare team.1
Five patients with history of stroke or vasculopathy were treated in the pivotal clinical trial (Study 1-C). All were at least 18 years old and on chronic transfusion therapy prior to LYFGENIA infusion. At 44-60 months follow-up, all five subjects remain transfusion independent without recurrent stroke.1
Limitations of Use: Following treatment with LYFGENIA, patients with α-thalassemia trait (-α3.7/-α3.7) may experience anemia with erythroid dysplasia that may require chronic red blood cell transfusions. LYFGENIA has not been studied in patients with more than two α-globin gene deletions.1
Efficacy & Safety of LYFGENIA
In Study 1-C, 88% (n=28/32) of patients treated with LYFGENIA in the pivotal clinical trial achieved the primary endpoint of complete resolution of [[VOEs]] (vaso-occlusive events) between 6 and 18 months after infusion. 94% (n=30/32) of patients treated in the same pivotal trial achieved the secondary endpoint of complete resolution of [[sVOEs]] (severe VOEs) between 6 and 18 months after infusion.1
After the primary evaluation period to last follow-up, 4 of 28 patients who achieved complete resolution of VOEs experienced VOEs.1
Study 1-C: The efficacy of LYFGENIA was studied in a single-arm, 24-month, open-label, multicenter Phase 1/2 study (Study 1-C) and a long-term follow-up study. In Study 1-C, 32 patients with a history of at least 4 VOEs in the 24 months prior to informed consent were evaluated for VOEs.1
The median (min, max) duration of follow-up for individuals infused with LYFGENIA (n=36) was 38 months (12, 61).1
No studies have been conducted to evaluate the effects of LYFGENIA on fertility. However, due to the use of chemotherapy during the conditioning process, gene therapy comes with a risk of infertility.1
The safety of LYFGENIA was evaluated in 45 patients. The most common adverse reactions ≥Grade 3 (incidence ≥20%) were stomatitis, thrombocytopenia, neutropenia, febrile neutropenia, anemia, and leukopenia.1
LYFGENIA has a boxed warning for hematologic malignancy. Hematologic malignancy has occurred in patients treated with LYFGENIA. Monitor patients closely for evidence of malignancy through complete blood counts at least every 6 months and through integration site analysis at Months 6, 12, and as warranted.1
The additional hematopoietic stress associated with mobilization, conditioning, and infusion of LYFGENIA, including the need to regenerate the hematopoietic system, may increase the risk of a hematologic malignancy.1
Patients with sickle cell disease have an increased risk of hematologic malignancy as compared to the general population. Patients treated with LYFGENIA may develop hematologic malignancies and should have lifelong monitoring.1
Treatment Process & Insurance Support
A patient’s out-of-pocket costs associated with LYFGENIA will depend on individual coverage policies and personal circumstances. Patients can reach out to Genetix Patient Support for a comprehensive overview of the anticipated costs they can expect relative to their out-of-pocket maximum for their plan and the spend to date. This often includes details on out-of-pocket costs for a consult, inpatient hospital stay, and the therapy itself. Patients can enroll in the Genetix Patient Support Program for this information without being prescribed LYFGENIA.
After enrolling in the Genetix Patient Support Program, patients can connect with a Patient Navigator.
LYFGENIA is only available at a Qualified Treatment Center (QTC). A QTC is a specialized hospital qualified to administer a Genetix Biotherapeutics gene therapy. Each LYFGENIA QTC has been carefully selected based on its expertise in areas such as transplant, cell and gene therapy, and sickle cell disease, and is trained to administer LYFGENIA.
If you’re interested in a [[Qualified Treatment Center (QTC)]], please access our QTC locator tool. The QTC locator tool is updated as new QTCs are added to the LYFGENIA network.