In the fourth quarter of 2023, the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) approved more than 20 new oncology agents and 16 new indications for previously approved agents. The approvals were for approximately 24 solid tumor types and 8 hematologic malignancies. There were 2 diagnostic agents, 5 biologics, 4 programmed cell death protein 1 inhibitors, and drugs with several other targets approved. One treatment was approved by both the FDA and the EMA: pembrolizumab (Keytruda®; Merck & Co) for treatment of gastric or gastroesophageal junction (GEJ) adenocarcinoma.
Additional highlights from the fourth quarter of 2023
- First FDA approval of a therapy to treat progressing desmoid tumors: nirogacestat (Ogsiveo™; SpringWorks Therapeutics)
- First FDA approval of a therapy intended to reduce the risk of relapse in pediatric patients with high-risk neuroblastoma: eflornithine (Iwilfin™; USWM)
- First FDA approval that includes patients with ROS proto-oncogene 1 (ROS1)-positive non-small cell lung cancer (NSCLC) who have previously received a ROS1 tyrosine kinase inhibitor (TKI), in addition to patients who are TKI naive
- Pembrolizumab (Keytruda; Merck & Co) had 7 new approvals; 5 by the FDA, 1 by the EMA, and 1 by both agencies
- There was also an update to the gastric or GEJ adenocarcinoma indication for pembrolizumab (Keytruda; Merck & Co) by the FDA that restricts use to patients whose tumors express programmed cell death ligand 1 (PD-L1) (combined positive score ³1) as determined by an FDA-approved test
- A combination of 2 products that previously received accelerated approval gained full approval by the FDA: enfortumab vedotin (Padcev®; Astellas) and pembrolizumab (Keytruda; Merck & Co), for urothelial carcinoma
- 1 new supportive care biologic agent was approved by the FDA: efbemalenograstim alfa-vuxw (Ryzneuta®; Evive Biotech), for prevention of chemotherapy-induced neutropenia
- 4 companion diagnostics were approved by the FDA
- FoundationOne® companion diagnostic for patients with NSCLC who may be eligible for combination treatment with binimetinib (Mektovi®; Array Biopharma) and encorafenib (Braftovi®; Array Biopharma)
- FoundationOne companion diagnostic for patients with hormone receptor-positive, human epidermal growth factor receptor 2-negative breast cancer who may be eligible for treatment with capivasertib (Truqap™; AstraZeneca)
- Abbott RealTime IDH1 assay to select patients appropriate for ivosidenib (Tibsovo®; Servier)
- Agilent PD-L1 IHC 22C3 pharmDx to select patients with gastric or GEJ adenocarcinoma appropriate for treatment with pembrolizumab (Keytruda; Merck & Co)
- 2 formulations of gadopiclenol, an agent for contrast-enhanced magnetic resonance imaging, received positive opinion by the EMA: Elucirem™(Guerbet) and Vueway™(Bracco Imaging), to detect lesions in the central nervous system and other body regions (eg, breast, liver, prostate)
- Under its Project Orbis initiative, the FDA collaborated with other health agencies to grant 7 approvals. Since its launch in 2019, there has been continued growth in the number of applications submitted to Project Orbis; EMA announced plans to join the multicountry review consortium as an observer
- 6 FDA approvals used the Real-Time Oncology Review (RTOR) pilot program, which streamlined data submission prior to the filing of the entire clinical application: nivolumab (Opdivo®; Bristol Myers Squibb), eflornithine (Iwilfin
- Re-examination of adagrasib (Krazati®; Mirati Therapeutics) by the EMA resulted in a conditional marketing authorization
- Non-renewal of the conditional marketing authorization for belantamab mafodotin (Blenrep; GSK Ireland) in multiple myeloma was confirmed by the EMA
The tables below summarize key Q4 2023 oncology approvals.
FDA and EMA Approvals
|
Generic Name
|
Brand Name
|
Tumor Typea
|
MOA
|
Manufacturer
|
Approval
|
Pembrolizumabb
|
Keytruda®
|
Gastric or GEJ adenocarcinoma
|
PD-1 inhibitor
|
Merck & Co
|
811/16/23 (FDA) and October 2023 (EMA)e
|
FDA Approvals
|
Generic Name
|
Brand Name
|
Tumor Typea
|
MOA
|
Manufacturer
|
Approval
|
Avelumabb
|
Bavencio
|
Merkel cell carcinoma
|
PD-L1 inhibitor
|
EMD Serono
|
9/6/23
|
Bosutinib monohydrate
|
Bosulif
|
Pediatric Ph+ CML
|
Tyrosine kinase inhibitor
|
PF Prism CV
|
9/26/23
|
Dabrafenib
|
Tafinlar
|
Tumor agnostic
|
B-Raf inhibitor
|
Novartis
|
8/31/23
|
Dostarlimab-gxlyb
|
Jemperli
|
dMMR endometrial cancer
|
PD-1 inhibitor
|
GlaxoSmithKline
|
7/31/23
|
Elranatamab-bcmmb
|
Elrexfio
|
Multiple myeloma
|
Bispecific antibody
|
Pfizer
|
8/14/23c
|
Fosaprepitant
|
Focinvez
|
Chemotherapy-induced nausea and vomiting
|
Antiemetic
|
Spes Pharmaceuticals
|
8/22/23
|
Luspatercept
|
Reblozyl
|
Anemia in patients with MDS
|
Recombinant fusion protein
|
Bristol Myers Squibb
|
8/28/23
|
Melphalan
|
Hepzato Kit
|
Uveal melanoma with unresectable hepatic metastases
|
Alkylating agent
|
Delcath Systems
|
8/14/23
|
Momelotinib
|
Ojjaara
|
Myelofibrosis and anemia
|
JAK1/JAK2 and activin A receptor type 1 inhibitor
|
GlaxoSmithKline
|
9/15/23
|
Motixafortide
|
Aphexda™
|
Stem cell mobilization in patients with multiple myeloma
|
CXCR4 antagonist
|
BioLineRx
|
9/8/23
|
Niraparib-abiraterone acetate
|
Akeega
|
Prostate cancer
|
PARP inhibitor plus abiraterone acetate
|
Janssen Biotech
|
8/11/23
|
Pralsetinib
|
Gavreto®
|
NSCLC
|
RET inhibitor
|
Genentech
|
8/20/23
|
Temozolomide
|
Temodar
|
Anaplastic astrocytoma
|
Alkylating agent
|
Merck
|
9/14/23
|
Therascreen PDGFRA RGQ PCR Kit
|
Therascreen PDGFRA Kit
|
GI tumors; companion diagnostic for avapritinib
|
PDGFRA assay
|
Qiagen
|
8/7/23
|
Trametinib
|
Mekinist
|
Tumor agnostic, age 1 and older
|
MEK inhibitor
|
Novartis
|
8/31/23
|
N/A
|
FoundationOne CDx
|
Solid tumors
|
Tissue-based diagnostic
|
Foundation Medicine
|
8/14/23
|
EMA Approvals
|
Generic Name
|
Brand Name
|
Tumor Typea
|
MOA
|
Manufacturer
|
Approval
|
Brentuximab vedotin
|
Adcetris
|
Stage III Hodgkin lymphoma
|
Antibody-drug conjugate
|
Takeda Pharmaceutical
|
September 2023
|
Crisantaspase
|
Enrylaze®
|
ALL and lymphoblastic lymphoma
|
Recombinant Erwinia asparaginase or crisantaspase
|
Jazz Pharmaceuticals
|
July 2023e
|
Dabrafenib
|
Finlee®
|
Glioma
|
B-Raf inhibitor
|
Novartis
|
September 2023e
|
Decitabine- cedazuridine
|
Inqovi®
|
AML
|
Hypomethylating agent
|
Otsuka Pharmaceuticals
|
July 2023
|
Degarelix acetate (generic)
|
Degarelix Accord
|
Prostate cancer
|
GnRH antagonist
|
Accord
|
July 2023
|
Elacestrant
|
Orserdu™
|
ER+, HER2– breast cancer with ESR1 mutation
|
ER antagonist
|
Stemline Therapeutics
|
July 2023e
|
Epcoritamab
|
Tepkinly®
|
DLBCL
|
Bispecific antibody
|
AbbVie
|
July 2023e
|
Glofitamab
|
Columvi®
|
DLBCL
|
Bispecific antibody
|
Roche
|
July 2023d
|
Melphalan flufenamide
|
Pepaxti
|
Multiple myeloma
|
XPO1 inhibitor
|
Oncopeptides AB
|
September 2023
|
Nivolumab
|
Opdivo
|
Melanoma
|
PD-1 inhibitor
|
Bristol Myers Squibb
|
July 2023e
|
Pembrolizumab
|
Keytruda
|
NSCLC (adjuvant therapy)
|
PD-1 inhibitor
|
Merck Sharp & Dohme
|
September 2023
|
Pembrolizumab
|
Keytruda
|
HER2+ gastric or gastroesophageal junction adenocarcinoma
|
PD-1 inhibitor
|
Merck Sharp & Dohme
|
July 2023e
|
Piflufolastat (18F)
|
Pylclari
|
Prostate cancer diagnosis
|
Diagnostic solution
|
Curium
|
July 2023
|
Sacituzumab govitecan
|
Trodelvy
|
HR+, HER2– metastatic breast cancer
|
Trop-2–directed ADC
|
Gilead
|
July 2023
|
Tislelizumab
|
Tevimbra
|
Esophageal squamous cell carcinoma
|
PD-1 inhibitor
|
Novartis
|
July 2023e
|
Trastuzumab (biosimilar)
|
Herwenda®
|
Breast cancer and gastric cancer
|
Monoclonal antibody
|
Sandoz GmbH
|
September 2023e
|
Trastuzumab deruxtecan
|
Enhertu
|
NSCLC with HER2 mutation
|
Antibody-drug conjugate
|
Daiichi Sankyo
|
September 2023
|
+, positive; –, negative; AKT, protein kinase B; ALL, acute lymphocytic leukemia; BRAF, v-Raf murine sarcoma viral oncogene homolog B; BTK, Bruton tyrosine kinase; CLL, chronic lymphocytic leukemia; CML, chronic myeloid leukemia; EMA, European Medicines Agency; FDA, US Food and Drug Administration; G-CSF, granulocyte colony-stimulating factor; GEJ, gastroesophageal junction; HER2, human epidermal growth factor receptor 2; HR, hormone receptor; IDH1, isocitrate dehydrogenase 1; JAK, Janus kinase; KRAS, Kirsten rat sarcoma viral proto-oncogene; MDS, myelodysplastic syndrome; MEK, mitogen-activated ERK kinase; MOA, mechanism of action; NSCLC, non-small cell lung cancer; PARP, poly(ADP-ribose) polymerase; PD-1, programmed cell death protein 1; PD-L1, PD-1 ligand 1; PDGFRA, platelet-derived growth factor receptor alpha; Ph, Philadelphia chromosome; RCC, renal cell carcinoma; ROS1, ROS proto-oncogene 1; SLL, small lymphocytic lymphoma; TKI, tyrosine kinase inhibitor; TRK, tropomyosin receptor kinase; VEGF, vascular endothelial growth factor.
aPlease see each product’s prescribing information and summary of product characteristics for full indications and labeling information.
bBiologic.
cAccelerated approval.
dConditional marketing authorization.
ePositive opinion.
|
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.raps.org/News-and-Articles/News-Articles/2023/11/FDA-notes-continued-growth”-of-Orbis-collaborative |
