The U.S. Food and Drug Administration (FDA) has recently granted approval to tisagenlecleucel, which is marketed under the name Kymriah, for adult patients with relapsed or refractory (R/R) follicular lymphoma who have already received two prior treatments. This decision came after the regulatory agency reviewed the results of the ELARA trial.
About Follicular Lymphoma
First thing’s first, let’s go over what this type of lymphoma is. Follicular lymphoma is a form of non-Hodgkin’s lymphoma, and it is typically a slow-growing cancer. It tends to impact those above the age of 60 and accounts for one of every five cases of lymphoma. This cancer occurs when the DNA of B-cells mutates, making them grow and divide rapidly. These abnormal cells then crowd out the healthy ones, causing the characteristic symptoms and lowering the body’s ability to fight infections.
- Night sweats
- Swollen lymph nodes
- Shortness of breath
- Weight loss
About FDA Approval
FDA approval would not have been possible without the positive results of the ELARA trial, so let’s dive into it. Titled ELARA, this phase 2 trial was international, single-center, and enrolled 98 R/R follicular lymphoma patients over the age of 18. All of these patients had a choice to begin on bridging chemotherapy before they were treated with restaging and lymphodepletion chemotherapy. This was then followed by tisagenlecleucel treatment. Doses ranged from 0.6 x 108 CAR-positive viable T cells to 6.0 x 108 cells.
Researchers focused on the primary endpoint of complete response (CR) assessed by an independent review committee (IRC) and the secondary endpoints of progression-free survival (PFS), objective response rate (ORR), duration of response (DOR), cellular kinetics, and overall survival (OS). Results include:
- ORR of 86.2%
- CR of 66.0%
- 31 patients achieved a partial response
- 38.7% of these study participants saw this transition to a CR
- 6-month PFS stood at 76%
- Both the median DOR and median time to the next anti-lymphoma therapy received were not reached
- Median OS and median PFS were not reached either
- 77.3% of adverse events (AEs) were attributed to treatment with tisagenlecleucel
- 28.9% of severe AEs were also attributed to tisagenlecleucel treatment
- Three people passed away during the trial, with all of the deaths connected to tisagenlecleucel
It was these results that led the FDA to approve this CAR-T cell therapy.
You can find the source article from OncLive.