The US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) approved 28 new or expanded indications for previously approved agents and 10 new oncology agents in the last quarter of 2024. The approvals included approximately 34 solid tumor types and 10 hematologic malignancies. More than two-thirds of the approvals were for biologics or biosimilars; there were 20 biologics and 2 biosimilars approved, 7 of which were new agents. One biologic treatment was approved by both the FDA and the EMA for a new indication: tislelizumab (Tevimbra®; BeiGene), a programmed death-ligand 1 (PD-L1)-blocking antibody approved for treatment of human epidermal growth factor receptor 2 (HER2)-negative gastric cancer or gastroesophageal junction (GEJ) adenocarcinoma.
Additional highlights from the fourth quarter of 2024
- The first medicine to treat von Hippel-Lindau disease, a rare genetic disorder causing cysts and tumors, was approved in Europe: belzutifan (Welireg™; Merck Sharp & Dohme), a hypoxia-inducible factor 2 alpha (HIF-2ɑ) inhibitor
- The first oral formulation of imatinib (Imkeldi; Shorla Oncology) received FDA approval
- The FDA approved a new formulation of nilotinib (Danziten™; Azurity) that is the first approved nilotinib formulation that does not require mealtime restrictions
- Non-small cell lung cancer (NSCLC) agents represent the highest number of approvals (7), followed by acute leukemia (5) and breast cancer (4)
- Two products that previously received accelerated approval gained full approval by the FDA
- Abemaciclib (Verzenio®; Eli Lilly and Company) in combination with an aromatase inhibitor as initial endocrine-based therapy for treatment of patients with advanced or metastatic breast cancer
- Pertuzumab, trastuzumab, and hyaluronidase-zzxf (Phesgo®; Genentech) for treatment of human epidermal growth factor receptor 2 (HER2)-positive breast cancer
- The most common molecular targets were programmed cell death protein 1 (PD-1; 5 approvals) and PD-L1 (4 approvals)
- There were 5 bispecific antibody approvals
- Five companion diagnostics were approved by the FDA
- FoundationOne® Liquid CDx (Foundation Medicine), a companion diagnostic for inavolisib, to identify patients with hormone receptor-positive, HER2-negative breast cancer with a phosphatidylinositol-4,5-bisphosphate 3-kinase catalytic subunit alpha (PIK3CA) mutation
- VENTANA® Pathway anti-HER2/neu (4B5) Rabbit Monoclonal Primary Antibody Assay (Ventana Medical Systems, Inc/Roche Diagnostics), a companion diagnostic for zanidatamab-hrii (Ziihera®; Jazz Pharmaceuticals) to identify patients with HER2-positive, immunohistochemistry (IHC) 3-positive biliary tract cancer
- VENTANA CLDN18 (43-14A) RxDx Assay (Ventana Medical Systems, Inc/Roche Diagnostics), a companion diagnostic for treatment with zolbetuximab-clzb (Vyloy®; Astellas Pharma) to identify patients with HER2-negative gastric or GEJ adenocarcinoma whose tumors are claudin 18.2 positive
- Ion Torrent™ Oncomine™ Dx Target Test (Thermo Fisher Scientific), a companion diagnostic for vorasidenib (Voranigo®; Servier Pharmaceuticals), to determine isocitrate dehydrogenase mutation status in patients with glioma
- MI Cancer Seek™ (Caris Life Sciences), a companion diagnostic to identify patients who may benefit from treatment with targeted therapies for breast cancer, colorectal cancer, melanoma, NSCLC, solid tumors, or endometrial carcinoma
- Two wearable devices received FDA approval
- Optune Lua® (Novocure; approved October 15), a first-of-its-kind wearable device for use concurrently with PD-1/PD-L1 inhibitors or docetaxel in patients with metastatic NSCLC
- Delivers Tumor Treating Fields (TTFields), which exert physical forces on the electrically charged components of dividing cancer cells, resulting in cell death
- On November 21, 2024, FDA approved new Head Flexible Electrode (HFE) transducer arrays for use with Optune Gio® (Novocure), another wearable portable device that produces TTFields, for treatment of glioblastoma multiforme
- Patients will be converted from Optune Gio to the new HFE arrays through the first half of 2025 through a controlled transition plan
- Two noteworthy FDA approvals of generics include the first generic paclitaxel (Sandoz) and generic pazopanib
- Optune Lua® (Novocure; approved October 15), a first-of-its-kind wearable device for use concurrently with PD-1/PD-L1 inhibitors or docetaxel in patients with metastatic NSCLC
The tables below summarize key Q4 2024 oncology approvals. Treatments in bold font represent new agent approvals by each agency; all others are indication expansions or conversion from accelerated to full approval (FDA).
FDA and EMA Approvals |
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Generic Name |
Brand Name |
Tumor Typea |
MOA |
Manufacturer |
Approval |
Tislelizumabb |
Tevimbra® |
Gastric or GEJ adenocarcinoma |
PD-L1 inhibitor |
BeiGene |
12/26/24 (FDA)/October 2024c (EMA) |
FDA Approvals |
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Generic Name |
Brand Name |
Tumor Typea |
MOA |
Manufacturer |
Approval |
Abemaciclib |
Verzenio® |
Breast |
CDK4/6 inhibitor |
Eli Lilly and Company |
11/13/24 |
Asciminib |
Scemblix® |
Ph+ CML (chronic phase) |
ABL/BCR-ABL1 inhibitor |
Novartis |
10/29/24d |
Cosibelimab-ipdlb |
Unloxcyt™ |
CSCC |
PD-L1 inhibitor |
Checkpoint Therapeutics |
12/13/24 |
Durvalumabb |
Imfinzi® |
Limited-stage SCLC |
PD-L1 inhibitor |
AstraZeneca |
12/4/24 |
Encorafenib |
Braftovi® |
Colorectal |
BRAF inhibitor |
Array BioPharma |
12/20/24d |
Ensartinib |
Ensacove™ |
NSCLC |
ALK inhibitor |
Xcovery Holdings |
12/18/24 |
Filgrastimb |
Zarxio® |
For patients acutely exposed to myelosuppressive doses of radiation |
G-CSF |
Sandoz |
10/22/24 |
Imatinib |
Imkeldi |
CML, ALL, MDS/
|
TKI |
Shorla Oncology |
11/22/24 |
Inavolisib |
Itovebi™ |
Breast |
PI3K inhibitor |
Genentech |
10/10/24 |
Methotrexate |
Jylamvo™ |
ALL |
Antimetabolite |
Shorla Oncology |
10/28/24 |
Nilotinib |
Danziten™ |
Ph+ CML |
TKI |
Azurity |
11/7/24 |
Nivolumabb |
Opdivo® |
NSCLC (combination, then monotherapy) |
PD-1 inhibitor |
Bristol Myers Squibb |
10/3/24 |
Nivolumab and hyaluronidase-nvhyb |
Opdivo Qvantig™ |
Multiple solid tumors |
PD-1 inhibitor |
Bristol Myers Squibb |
12/27/24 |
Obecabtagene autoleucelb |
Aucatzyl® |
ALL |
CAR T-cell therapy |
Autolus |
11/8/24 |
Pertuzumab, trastuzumab,
|
Phesgo® |
Breast |
Monoclonal antibodies and endoglycosidase |
Genentech |
11/22/24 |
Revumenib |
Revuforj® |
Acute leukemia with KMT2A translocation |
Menin inhibitor |
Syndax |
11/15/24 |
Zanidatamab-hriib |
Ziihera® |
Biliary tract |
HER2-directed bispecific antibody |
Jazz Pharmaceuticals |
11/20/24d |
Zeno
|
Bizengri® |
NSCLC and pancreatic adenocarcinoma |
HER2- and HER3-directed bispecific antibody |
Merus N.V. |
12/4/24d |
Zolbetuximab-clzbb |
Vyloy® |
Gastric or GEJ adenocarcinoma |
Claudin 18.2-directed cytolytic antibody |
Astellas Pharma |
4/25/24 |
EMA Approvals |
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Generic Name |
Brand Name |
Tumor Typea |
MOA |
Manufacturer |
Approval |
Amivantamab-vmjwb |
Rybrevant® |
NSCLC |
Bispecific antibody |
Janssen-Cilag |
November 2024c |
Belzutifanb |
Welireg™ |
von Hippel-Lindau disease-related tumors and clear cell RCC |
HIF-2ɑ inhibitor |
Merck Sharp & Dohme |
December 2024e |
Blinatumomabb |
Blincyto® |
ALL |
Bispecific antibody |
Amgen Europe |
December 2024c |
Catumaxomabb |
Korjuny® |
Malignant ascites |
Bispecific antibody |
Lindis Biotech |
October 2024c |
Denosumabf |
Osenvelt® |
Advanced cancer involving bone and giant cell bone tumor |
RANKL-targeted monoclonal antibody |
Celltrion Healthcare |
December 2024c |
Denosumabf |
Xbryk |
Advanced cancer involving bone and giant cell bone tumor |
RANKL-targeted monoclonal antibody |
Samsung Bioepis |
November 2024c |
Dostarlimab-gxlyb |
Jemperli |
Endometrial |
PD-1 inhibitor |
GlaxoSmithKline (Ireland) |
December 2024c |
Filgrastimf |
Zefylti |
Neutropenia and mobilization of peripheral blood progenitor cells |
G-CSF |
CuraTeQ Biologics |
December 2024c |
Ipilimumabb |
Yervoy® |
Colorectal |
CTLA-4 inhibitor |
Bristol Myers Squibb |
November 2024c |
Isatuximab-irfcb |
Sarclisa® |
Multiple myeloma (combination) |
Anti-CD38 monoclonal antibody |
Sanofi Winthrop Industrie |
November 2024c |
Lazertinib |
Lazcluze® |
NSCLC |
EGFR TKI |
Janssen-Cilag |
November 2024c |
Nivolumabb |
Opdivo® |
Colorectal |
PD-1 inhibitor |
Bristol Myers Squibb |
November 2024c |
Osimertinib |
Tagrisso® |
NSCLC |
EGFR TKI |
AstraZeneca |
November 2024c |
Pembrolizumabb |
Keytruda® |
Mesothelioma |
PD-1 inhibitor |
Merck Sharp & Dohme |
November 2024c |
Repotrectinib |
Augtyro™ |
ROS1+ advanced solid tumors or NSCLC; solid tumors |
ROS1 and TRK inhibitor |
Bristol Myers Squibb |
November 2024e |
Ribociclibb |
Kisqali® |
Early breast cancer |
CDK4/6 inhibitor |
Novartis |
October 2024c |
Tislelizumabb |
Tevimbra® |
Esophageal squamous cell carcinoma |
PD-L1 inhibitor |
BeiGene |
October 2024c |
aPlease see each product’s prescribing information and summary of product characteristics for full indications and labeling information.bBiologic.cPositive opinion.dAccelerated approval.eConditional marketing authorization.fBiosimilar.ABL/BCR-ABL1, breakthrough cluster region/ABL proto-oncogene 1; ALK, anaplastic lymphoma kinase; ALL, acute lymphocytic leukemia; BRAF, v-raf murine sarcoma viral oncogene homolog B1; CAR, chimeric antigen receptor; CDK4/6, cyclin-dependent kinase 4/6; CML, chronic myeloid leukemia; CSCC, cutaneous squamous cell carcinoma; CTLA-4, cytotoxic T-lymphocyte-associated antigen 4; EGFR, epidermal growth factor receptor; EMA, European Medicines Agency; FDA, US Food and Drug Administration; G-CSF, granulocyte colony-stimulating factor; GEJ, gastroesophageal junction; GIST, gastrointestinal stromal tumor; HER2, human epidermal growth factor receptor 2; HIF-2ɑ, hypoxia-inducible factor 2 alpha; KMT2A, lysine methyltransferase 2A; MDS, myelodysplastic syndromes; MOA, mechanism of action; NSCLC, non-small cell lung cancer; PD-1, programmed cell death protein 1; PD-L1, programmed death-ligand 1; Ph, Philadelphia chromosome; PI3K, phosphoinositide 3-kinase; PIK3CA, phosphatidylinositol-4,5-bisphosphate 3-kinase catalytic subunit alpha; RANKL, receptor activator of nuclear factor kappa beta; RCC, renal cell carcinoma; ROS1, ROS proto-oncogene 1; SCLC, small cell lung cancer; TKI, tyrosine kinase inhibitor; TRK, tropomyosin receptor kinase. |
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Sources:
https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=reportsSearch.process
https://www.ema.europa.eu/en/committees/chmp/chmp-agendas-minutes-highlights
