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 |
