CAR T-Cell FDA Approval: Status Update
In addition to the approval of Kymriah, Yescarta (axicabtagene ciloleucel or Axi-Cel) was approved on October 18, 2017 for the treatment of adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy. Data from Kite’s ZUMA-1 trial supported the new drug application (NDA).
Clinical trial results with Yescarta have been remarkable in refractory leukemias and lymphomas, leading to sustained, high rates of remission in a large percentage of treated patients.
However, treatments are not without serious risks and the manufacturing process is complicated and lengthy. Side effects, which can be difficult for the patient, can be managed by physicians in most cases. But it’s important to remember when thinking about risks – these patients usually have limited or no other treatment options.
What is Yescarta?
Axicabtagene ciloleucel (Yescarta) from Kite Pharma, and initially developed at the National Cancer Institute (NCI), is an anti-CD19 CAR-T therapy. Yescarta is under study or approved for various relapsed and refractory hematologic malignancies, including:
- Now approved – Aggressive B-cell non-Hodgkin lymphoma (diffuse large b-cell lymphoma, primary mediastinal b-cell lymphoma, and transformed follicular lymphoma) (ZUMA-1 trial) – this pivotal clincial trial is currently at the FDA undergoing regulatory review for approval for NHL.
- Relapsed/Refractory Mantle Cell Lymphoma (ZUMA-2 trial)
- Chronic Lymphocytic Leukemia (ZUMA-8 trial)
- Adult and Pediatric Relapsed/Refractory Acute Lymphoblastic Leukemia (ZUMA-3 and ZUMA-4 trials)
Yescarta Approval for Diffuse Large B-cell Lymphoma
Approximately 72,000 new cases of non-Hodgkin lymphoma (NHL) are diagnosed in the U.S. each year, and diffuse large B-cell lymphoma (DLBCL) represents approximately 33% of newly diagnosed cases.
Yescarta is approved for use in adult patients with large B-cell lymphoma after at least two other kinds of treatment failed. Subtypes of large B-cell lymphoma include:
– diffuse large B-cell lymphoma (DLBCL)
– primary mediastinal large B-cell lymphoma
– high grade B-cell lymphoma
– DLBCL arising from follicular lymphoma
Yescarta is not indicated for the treatment of patients with primary central nervous system lymphoma.
Diffuse large B-cell lymphoma (DLBCL) is the most common type of NHL in adults. NHLs are cancers that begin in certain cells of the immune system and can be either fast-growing (aggressive) or slow-growing.
The European Union submission of axicabtagene ciloleucel is expected in the 3rd quarter of 2017.
