Trailblazing trial design: LYFGENIA™ is the result of the longest-studied gene therapy program in SCD1,2
LYFGENIA was evaluated in 2 clinical trials1
LYFGENIA was evaluated in 2 clinical trials1
The safety of LYFGENIA was based on patients with sickle cell disease in 1 open-label, single-arm clinical trial (Study 1: HGB-206) and 1 long-term follow-up study (LTF-307).1,3 Study 1 started in February 2015.
The efficacy of LYFGENIA was studied in a single-arm, 24-month, open-label, multicenter Phase 1/2 study (Study 1-C).1
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
LYFGENIA Safety and Efficacy
See experts lead an in-depth review of the clinical trial, efficacy data, and safety information for LYFGENIA
LYFGENIA Safety and Efficacy
See experts lead an in-depth review of the clinical trial, efficacy data, and safety information for LYFGENIA
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Primary Endpoint Post-infusion Stroke Outcomes Data Globin Response
Primary Endpoint
Post-infusion
Stroke Outcomes Data
Globin Response
LYFGENIA is a one-time transformational SCD gene therapy with the potential to decrease or stop VOEs.
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
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
Primary Endpoint1
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
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
Secondary Endpoint1
Follow-up Time1
-
LYFGENIA is the only approved SCD gene therapy supported by a study design that used a fixed efficacy assessment window that was predefined, ensuring patients were evaluated for (s)VOEs between 6-18 months after infusion.1,4
LYFGENIA is the only approved SCD gene therapy supported by a study design that used a fixed efficacy assessment window that was predefined, ensuring patients were evaluated for (s)VOEs between 6-18 months after infusion.1,4
LYFGENIA delivered powerful and lasting reduction in VOEs
Vaso-occlusive events before and after LYFGENIA infusion5
sVOEs were also counted as VOEs.
This figure represents patient-level data and is not included in the USPI.
Vaso-occlusive events
before and after LYFGENIA
infusion4
sVOEs were also counted as VOEs.
This figure represents patient-level data and is not included in the USPI.
Vaso-Occlusive Events (VOEs)1
In the LYFGENIA study, 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 hours
- acute chest syndrome
- acute hepatic sequestration
- acute splenic sequestration
Severe Vaso-Occlusive Events (sVOEs)1
In the LYFGENIA study, severe vaso-occlusive events (sVOEs) were defined as vaso-occlusive events (VOEs) requiring either of the following:
- a hospitalization
- multiple visits to an emergency department/urgent care over 72 hours and receiving IV medications at each visit
- priapism requiring any level of medical attention
STUDY 1-C
Demonstrated resilience in stroke patients — 5 patients remained stroke-free after LYFGENIA
Demonstrated resilience in stroke patients — 5 patients remained stroke-free after LYFGENIA
Five patients (≥18 years of age) in Study 1-C with a history of stroke or vasculopathy who were on chronic transfusion therapy prior to LYFGENIA infusion remained transfusion-independent and without recurrent stroke at 44–60 months follow-up.1
The only approved SCD gene therapy with stroke outcome data included in the Prescribing Information1,4
Stable. Durable. Proven.
LYFGENIA maintained stable and durable HbAT87Q levels from month 6 through month 48.1
All 36 patients infused in Study 1-C (transplant population) were evaluated for globin response. 31/36 (86%) achieved globin response. All patients who achieved globin response maintained it.1
Globin response was defined as meeting the following criteria for a continuous period of at least 6 months after drug product infusion:
- weighted average hemoglobin A percentage of non-transfused total Hb ≥30% AND1
- weighted average non-transfused total Hb (HbS + HbF + HbA2 + HbAT87Q) increase of ≥3 g/dL compared to baseline total Hb OR weighted average non-transfused total Hb ≥10 g/dL.1
Achieved Globin Response1
(n=31/36)
All members of the transplant population (n=36) were evaluated for globin response
Achieved Globin Response1
(n=31/36)
All members of the transplant population (n=36) were evaluated for globin response
Patients Who
Achieved Globin Response
Maintained It1
(n=31/31)
Patients Who
Achieved Globin Response &
Maintained It1
(n=31/31)
Globin response, a secondary endpoint of the LYFGENIA clinical trials, was defined as meeting the following criteria for a continuous period of at least 6 months after LYFGENIA infusion:
- Weighted average hemoglobin AT87Q percentage of non-transfused total Hb ≥30% AND1
- Weighted average non-transfused total Hb (HbS + HbF + HbA2 + HbAT87Q) increase of ≥3 g/dL compared to baseline total Hb OR weighted average non-transfused total Hb ≥10 g/dL1
Median concentration of total Hb after LYFGENIA infusion6
Median concentration of total Hb after LYFGENIA infusion6
Total Hb consists of HbS, HbAT87Q, fetal hemoglobin (HbF), and HbA2
Other Hb includes HbS, HbF, and HbA2
This data is from a secondary efficacy endpoint; however, there was no formal hypothesis testing.
At 36 months (n=20), more than half of patients reported clinically meaningful improvements in the following measures7:
This exploratory analysis includes a subset of adult Study 1-C patients with available baseline and follow-up health-related quality of life (HRQOL) data (PROMIS-57).7
No efficacy conclusions should be drawn from these data.
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