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How LYFGENIA was studied

The safety of LYFGENIA was studied in individuals with sickle cell disease in 1 clinical trial and 1 long-term follow-up study.

 

The clinical trial
included:

54

people

who initiated stem cell collection†

Individuals with a
median age of

25

years

(min age 12 years, max age 43 years)

The median duration of
follow-up for individuals
treated with LYFGENIA†:

42

months

(min 12 months; max 87 months)

†45 people were treated with LYFGENIA.

A one-time transformational gene therapy with the potential to decrease or stop vaso-occlusive events (VOEs)

A one-time transformational gene therapy with the potential to decrease or stop vaso-occlusive events (VOEs)

The efficacy of LYFGENIA was studied in 36 individuals. 32 individuals were evaluated for the number of
[[vaso-occlusive events (VOEs)]] they experienced between 6–18 months after treatment.

88 percent of individuals

did not experience any [[vaso-occlusive events (VOEs)]]

(28/32 individuals)

94 percent of individuals

did not experience any [[severe vaso-occlusive events (sVOEs)]]

(30/32 individuals)

What else should I know about the study?

4 individuals experienced vaso-occlusive events (VOEs) after 18 months. These individuals did not experience any VOEs between 6–18 months after treatment
No clinically meaningful differences in efficacy and safety were observed between adults and adolescents
38 months was the median duration of follow-up for efficacy for the 36 individuals who received LYFGENIA (min of 12 months; max of 61 months)
5 individuals with a history of stroke or vasculopathy were treated. They were at least 18 years old and on chronic transfusion therapy prior to LYFGENIA infusion. At 44–60 months' follow-up, all individuals remain transfusion independent without repeated stroke

Vaso-occlusive events (VOEs) before and after LYFGENIA infusion

Graph: vaso-occlusive events before and after LYFGENIA

sVOEs were also counted as VOEs.

This figure is not included in the LYFGENIA Prescribing Information.

What are vaso-occlusive events (VOEs)?

In the LYFGENIA clinical study, vaso-occlusive events (VOEs) were defined as any of the following events, which need to be evaluated 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

What are severe vaso-occlusive events (sVOEs)?

In the LYFGENIA clinical study, severe vaso-occlusive events (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 visit
  • priapism requiring any level of medical attention
More topics to explore More topics to explore
Understanding Safety Information
The Steps to Treatment
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