In a year with many firsts observed, lisocabtagene maraleucel (liso-cel, Breyanzi) became the only chimeric antigen receptor (CAR) T-cell therapy to receive a stamp of approval for adults with relapsed or refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL). The agent also received 3 indications from the FDA in 2024, providing options for many different patient populations. Several CAR T-cell therapies also moved up in lines of therapy, providing an earlier cellular therapy for patients with select hematologic malignancies.

As the field expanded with almost 20 FDA approvals in the hematology sphere occurring in 2024, we’ve rounded up all the approvals and paired them with expert insights below!

Ponatinib (Iclusig) plus chemotherapy

Approval type: Accelerated
Indication: Adult patients with newly diagnosed Philadelphia chromosome (Ph)–positive acute lymphoblastic leukemia (ALL)
Month: March
To read more, visit:­ bit.ly/3VLjM4X

"We’re very excited about the completion of the [phase 3 PhALLCON trial (NCT03589326)] and meeting the primary end points, as well as the approval of ponatinib. As the treatment of [patients with] Ph-positive ALL continues to evolve, this approval introduces more effective agents early in the treatment paradigm, potentially sparing more patients from the need of treatment at the time of relapse.”

Ibrahim T. Aldoss, MD
City of Hope, Duarte, California

Inotuzumab ozogamicin (Besponsa)

Approval type: Regular
Indication: Pediatric patients aged 1 and older with relapsed or refractory CD22-positive B-cell precursor ALL
Month: March
To read more, visit: bit.ly/3VGExi8

Blinatumomab (Blincyto)

Approval type: Regular
Indication: Adult and pediatric patients 1 month or older who have CD19-positive, Ph-negative B-cell precursor ALL in the consolidation phase of multiphase chemotherapy
Month: June
To read more, visit: bit.ly/3ZYkZZt

Methotrexate (Jylamvo)

Approval type: Expanded
Indication: Expanded to include the treatment of pediatric patients with ALL and polyarticular juvenile idiopathic arthritis
Month: October
To read more, visit: bit.ly/3OvLr65

Asciminib (Scemblix)

Approval type: Accelerated
Indication: Adult patients with newly diagnosed Ph-positive chronic myeloid leukemia in chronic phase (CML-CP)
Month: October
To read more, visit: bit.ly/4fXL6oq

Obecabtagene autoleucel (obe-cel, Aucatzyl)

Approval type: Regular
Indication: Adult patients with relapsed or refractory B-cell precursor ALL
Month: November
To read more, visit: bit.ly/4gicCO0

"This approval comes at a critical time, and it’s a major milestone [for] patients with ALL. Splitting the dose allows us to deliver a safe CAR T-cell therapy [with] minimal complications, and [it is] effective in the long run. [Obe-cel is also] an outpatient therapy, which will have a major impact on society and our patients’ lives. It’s a very important accomplishment that will fill a major unmet need.”

Elias Jabbour, MD
The University of Texas MD Anderson Cancer Center, Houston

Revumenib (Revuforj)

Approval type: Regular
Indication: Adult and pediatric patients aged 1 and older with relapsed or refractory acute leukemia and a KMT2A translocation
Month: November
To read more, visit: bit.ly/49ImZbp

Zanubrutinib (Brukinsa) plus obinutuzumab (Gazyva)

Approval type: Accelerated
Indication: Relapsed or refractory follicular lymphoma after 2 or more lines of systemic therapy
Month: March
To read more, visit: bit.ly/3ByYrov

Liso-cel

Approval type: Accelerated
Indication: Adult patients with relapsed or refractory follicular lymphoma who have received 2 or more prior lines of systemic therapy
Month: May
To read more, visit: bit.ly/3VFiTuM

Liso-cel

Approval type: Regular
Indication: Adult patients with relapsed or refractory mantle cell lymphoma (MCL) who have received at least 2 prior lines of systemic therapy, including a Bruton tyrosine kinase (BTK) inhibitor
Month: May
To read more, visit: bit.ly/4gACd4o

Epcoritamab-bysp (Epkinly)

Approval type: Accelerated
Indication: Adult patients with relapsed or refractory follicular lymphoma following 2 or more lines of systemic therapy
Month: June
To read more, visit: bit.ly/3ZUYEMf

"[As] we have such an active therapy in the relapsed/refractory setting, [it] will [likely] move up in the lines of [treatment] in follicular lymphoma. We have studies [examining] second-line treatment and will be [evaluating] frontline therapy with bispecific antibodies, including epcoritamab…. I believe that bispecific antibodies, including epcoritamab, will let us develop chemotherapy-free combinations resulting in excellent efficacy, minimal toxicity, and very durable responses for patients, and hopefully one day [they may] cure follicular lymphoma.”

Grzegorz S. Nowakowski, MD
Mayo Clinic Comprehensive Cancer Center, Rochester, Minnesota

Denileukin diftitox-cxdl (Lymphir)

Approval type: Regular
Indication: Relapsed/refractory cutaneous T-cell lymphoma after at least 1 prior systemic therapy
Month: August
To read more, visit: bit.ly/41CU2LO

Liso-cel

Approval type: Accelerated
Indication: Adult patients with relapsed or
refractory CLL or SLL who have received at least 2 prior lines of therapy, including a BTK inhibitor and BCL2 inhibitor
Month: March
To read more, visit: bit.ly/4goj2eE

"There could be room for improvement with CAR T-cell therapy, and we’re working to figure out how to make [these agents] better in CLL. Nevertheless, having [an agent that can generate a] complete response in 1 in 5 patients who are heavily pretreated and don’t have effective options is quite significant.”

Saad J. Kenderian, MB, ChB
Mayo Clinic, Rochester, Minnesota

Idecabtagene vicleucel (Abecma)

Approval type: Regular
Indication: Adult patients with relapsed or refractory multiple myeloma after 2 or more prior lines of therapy, including an immunomodulatory drug (IMiD), a proteasome inhibitor (PI), and an anti-CD38 monoclonal antibody
Month: April
To read more, visit: bit.ly/4fiAfEF

Ciltacabtagene autoleucel (cilta-cel, Carvykti)

Approval type: Expanded
Indication: Adult patients with relapsed or refractory multiple myeloma who have received at least 1 prior line of therapy, including a PI and an IMiD, and whose disease is refractory to lenalidomide (Revlimid)
Month: April
To read more, visit:­ bit.ly/3ZJeSXk

"One thing that we have to recognize is the [limited] accessibility of CAR T-cell therapy as it stands, as well as the capacity at transplant cellular therapy centers. [We] also [should] appreciate the fact that we do have some triplet regimen options for patients in that early-relapsed setting. Having said all that, cilta-cel would be a very good option for the patients who are functionally high risk and who did not have a good first or even second progression-free survival period….The other important piece is that patients who do choose cilta-cel as an option [typically experience] a drug-free period after they’ve recovered from the CAR T-cell therapy. There are so many advantages, but we have to present those options with the caveat that logistics will be an issue.”

Saad Z. Usmani, MD, MBA, FACP, FASCO
Memorial Sloan Kettering Cancer Center, New York, New York

Isatuximab-irfc (Sarclisa) plus bortezomib (Velcade), lenalidomide, and dexamethasone (VRd)

Approval type: Regular
Indication: Adult patients with newly diagnosed multiple myeloma who are not eligible for autologous stem cell transplant (ASCT)
Month: September
To read more, visit: bit.ly/49Dj67H

Imetelstat (Rytelo)

Approval type: Regular
Indication: Adults with low- to intermediate-1–risk myelodysplastic syndromes (MDS) and transfusion-dependent anemia requiring 4 or more red blood cell units over 8 weeks who have not responded to, have lost response to, or are not eligible for erythropoiesis-stimulating agents
Month: June
To read more, visit: bit.ly/3Du6xiJ

"Following imetelstat’s approval for patients who are transfusion dependent with lower-risk MDS, we finally have another tool in our tool set for how to treat these patients. Findings [from the phase 3 IMerge trial (NCT02598661)] will help bolster [the] approval by demonstrating that not only do patients become transfusion independent, but they also have a rise in their hemoglobin levels irrespective of whether they have other abnormalities of other blood counts.”

Mikkael A. Sekeres, MD
University of Miami Sylvester Comprehensive Cancer Center, Florida

Daratumumab and hyaluronidase-fihj (Darzalex Faspro) plus VRd

Approval type: Regular
Indication: Induction and consolidation in patients with newly diagnosed multiple myeloma who are eligible for ASCT
Month: July
To read more, visit: bit.ly/3ZE7Xio

Remestemcel-L-rknd (Ryoncil)

Approval type: Regular
Indication: Pediatric patients 2 months and older with steroid-refractory acute graft-vs-host disease
Month: December
To read more, visit: bit.ly/3DFjp61

Reference

Advancing health through innovation: New drug therapy approvals 2024. FDA. Accessed February 4, 2025. bit.ly/3WKqf0s