The US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) started 2024 with more than 24 new or expanded indications for previously approved agents and 8 new oncology agents in the first quarter of the year. The approvals were for approximately 14 solid tumor types and 4 hematologic malignancies. Nearly 50% of the approvals were for biologics or biosimilars; there were 14 biologics approved, nearly half of which were programmed cell death protein 1 inhibitors, and 3 biosimilars. Two treatments were approved by both the FDA and the EMA: irinotecan liposome injection (Onivyde®; Ipsen), the first new agent approved for first-line treatment of metastatic pancreatic cancer in more than 10 years, and denosumab (Wyost®; Sandoz) for treatment of bone complications from cancer.
Additional highlights from the first quarter of 2024
- There were 5 approvals of treatments for non-small cell lung cancer (NSCLC)
- Pembrolizumab (Keytruda®; Merck Sharp & Dohme) had 3 new approvals: 2 by the FDA and 1 by the EMA
- 5 products that previously received accelerated approval gained full approval from the FDA
- Amivantamab-vmjw (Rybrevant®; Janssen Biotech) for NSCLC with epidermal growth factor receptor exon 20 insertion mutations
- Erdafitinib (Balversa®; Janssen Biotech) for urothelial carcinoma
- Mirvetuximab soravtansine-gynx (Elahere®; Immunogen/AbbVie) for epithelial ovarian, fallopian tube, or primary peritoneal cancer
- Tepotinib HCl (Tepmetko®; EMD Serono) for NSCLC
- Pembrolizumab (Keytruda; Merck) for hepatocellular carcinoma
- A new granulocyte colony-stimulating factor (G-CSF) biosimilar was approved by the FDA: pegfilgrastim-bmez (Ziextenzo®; Sandoz), for increased survival in patients acutely exposed to myelosuppressive doses of radiation
- One new G-CSF biologic agent received a positive opinion by the EMA: efbemalenograstim alfa-vuxw (Ryzneuta®; Evive Biotech), for prevention of chemotherapy-induced neutropenia (also approved by the FDA in Q4 2023)
- The FDA also approved a new artificial intelligence-powered, handheld prescription device for evaluation of skin lesions suggestive of melanoma, basal cell carcinoma, and/or squamous cell carcinoma
- The FDA added new safety information that pertains to the risk of transplant (including corneal graft) rejection to the labeling for the class of immune checkpoint inhibitors and a boxed warning on all chimeric antigen receptor T-cell therapies regarding risk of T-cell malignancies
- The FDA withdrew approval of melphalan flufenamide (Pepaxto®; Oncopeptides AB) for treatment of certain patients with multiple myeloma
The tables below summarize key Q1 2024 oncology approvals. Treatments in bold font represent new-agent approvals; all others are indication expansions or conversions from accelerated to full approvals (FDA).
FDA and EMA Approvals |
Generic Name | Brand Name | Tumor Typea | MOA | Manufacturer | Approval |
Denosumab (interchangeable biosimilar) | Wyost | Bone | RANKL inhibitor | Sandoz | 3/5/24 (FDA) and March 2024 (EMA)b |
Irinotecan liposome injection | Onivyde | Pancreatic | Liposomal chemotherapeutic | Ipsen | 2/13/24 (FDA) and March 2024 (EMA)b |
FDA Approvals | Generic Name | Brand Name | Tumor Typea | MOA | Manufacturer | Approval | Alpelisib | Piqray® | Breast cancer | PI3K inhibitor | Novartis | 1/18/24 | Amivantamab-vmjwc | Rybrevant | NSCLC | Bispecific antibody | Janssen Biotech | 3/1/24 | Cabazitaxel | Cabazitaxel | Prostate cancer | Microtubule inhibitor | Actavis | 3/14/24 | Denosumab-bbdz (interchangeable biosimilar) | Jubbonti® | Prostate and breast cancers (to increase bone mass) | RANKL inhibitor | Sandoz | 3/5/24 | Erdafitinib | Balversa | Urothelial carcinoma | FGFR inhibitor | Janssen Biotech | 1/19/24 | Inotuzumab ozogamicinc | Besponsa® | ALL | CD22-directed ADC | Wyeth Pharma | 3/6/24 | Lifileucelc | Amtagvi™ | Melanoma | T-cell immunotherapy | Iovance Biotherapeutics | 2/16/24d | Lisocabtagene maraleucelc | Breyanzi® | CLL and SLL | CAR T | Bristol Myers Squibb | 3/14/24d | Mirvetuximab soravtansine-gynxc | Elahere | Epithelial ovarian, fallopian tube, or primary peritoneal cancer | ADC | Immunogen, Inc (now AbbVie) | 3/22/24 | Nivolumabc | Opdivo® | Urothelial carcinoma | PD-1 inhibitor | Bristol Myers Squibb | 3/6/24 | Osimertinib | Tagrisso® | NSCLC | Kinase inhibitor | AstraZeneca | 2/16/24 | Pegfilgrastim-bmez (biosimilar) | Ziextenzo | Multiple (treated with chemotherapy) | G-CSF | Sandoz | 2/28/24 | Pembrolizumabc | Keytruda | Cervical cancer | PD-1 inhibitor | Merck | 1/12/24 | Pembrolizumabc | Keytruda | HCC | PD-1 inhibitor | Merck | 1/25/24 | Ponatinib HCl | Iclusig® | Ph+ ALL | BCR::ABL1 inhibitor | Takeda Pharmaceuticals | 3/19/24d | Talazoparib tosylate | Talzenna® | Breast cancer | PARP inhibitor | Pfizer | 3/7/24 | Tepotinib HCl | Tepmetko | NSCLC | MET inhibitor | EMD Serono | 2/15/24 | Tislelizumabc | Tevimbra® | Esophageal squamous cell carcinoma | PD-1 inhibitor | BeiGene | 3/13/24 | Zanubrutinib | Brukinsa® | Follicular lymphoma | BTK inhibitor | BeiGene | 8/14/23 | EMA Approvals | Generic Name | Brand Name | Tumor Typea | MOA | Manufacturer | Approval | Ciltacabtagene autoleucelc | Carvykti™ | Multiple myeloma | CAR T | Janssen-Cilag | February 2024b | Efbemalenograstim alfac | Ryzneuta | Multiple (neutropenia) | G-CSF | Evive Biotech | January 2024b | Enzalutamide | Xtandi® | Prostate cancer | Androgen receptor inhibitor | Astellas Pharma | March 2024b | Idecabtagene vicleucelc | Abecma® | Multiple myeloma | CAR T | Bristol Myers Squibb | January 2024e | Luspaterceptc | Reblozyl® | MDS (anemia) | PD-1 inhibitor | Bristol Myers Squibb | February 2024b | Pembrolizumabc | Keytruda | NSCLC | PD-1 inhibitor | Merck Sharp & Dohme | February 2024b | Retifanlimabc | Zynyz® | Merkel cell carcinoma | PD-1 inhibitor | Incyte | February 2024b | Selpercatinib | Retsevmo® | Thyroid cancer | RET inhibitor | Eli Lilly | January 2024e | Selpercatinib | Retsevmo® | Solid tumors | RET inhibitor | Eli Lilly | March 2024b | Tislelizumabc | Tizveni | NSCLC | PD-1 inhibitor | BeiGene | February 2024b | ABL1, ABL proto-oncogene 1; ADC, antibody-drug conjugate; ALL, acute lymphocytic leukemia; BCR, breakpoint cluster region; BTK, Bruton tyrosine kinase; CAR T, chimeric antigen receptor T cell; CLL, chronic lymphocytic leukemia; EMA, European Medicines Agency; FDA, US Food and Drug Administration; FGFR, fibroblast growth factor receptor; G-CSF, granulocyte colony-stimulating factor; HCC, hepatocellular carcinoma; MDS, myelodysplastic syndrome; MET, mesenchymal epithelial transition; MOA, mechanism of action; NSCLC, non-small cell lung cancer; PARP, poly(ADP-ribose) polymerase; PD-1, programmed cell death protein 1; Ph+, Philadelphia chromosome-positive; PI3K, phosphoinositide 3-kinase; RANKL, receptor activator of nuclear factor kappa beta ligand; RET, rearranged during transfection; SLL, small lymphocytic lymphoma. aPlease see each product’s prescribing information and summary of product characteristics for full indications and labeling information. bPositive opinion. cBiologic. dAccelerated approval. eMarketing authorization. | 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 https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications https://www.fda.gov/news-events/press-announcements/fda-roundup-january-16-2024 https://www.reuters.com/business/healthcare-pharmaceuticals/us-fda-requires-boxed-warning-car-t-cancer-therapies-2024-01-23/ https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/breyanzi-lisocabtagene-maraleucel https://www.fda.gov/drugs/drug-safety-and-availability/fda-issues-final-decision-withdraw-approval-pepaxto-melphalan-flufenamide?utm_medium=email&utm_source=govdeliveryPepaxto https://www.fda.gov/news-events/press-announcements/fda-roundup-march-5-2024 |
