New Treatment Options in Oncology: FDA and EMA Drug Approvals in Q3 2023 – Broad Scope of Unique Targets and Malignancies

In the third quarter of 2023, the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) approved more than 36 new oncology agents and new indications for previously approved agents. The cancer types with the most approvals were multiple myeloma, breast cancer, prostate cancer, and non-small cell lung cancer (NSCLC). There were 4 diagnostic agents, 4 biologics, 4 programmed cell death protein 1 inhibitors, 4 bispecific antibodies, and 3 antibody-drug conjugates approved, with 9 indication expansions for existing agents. Three treatments were approved by both the FDA and the EMA: quizartinib (Vanflyta^®; Daiichi Sankyo), talquetamab-tgvs (Talvey™; Janssen Biotech), and trifluridine/tipiracil (Lonsurf^®; Taiho Oncology [USA] and Servier [France]), which received an indication expansion from monotherapy to combination use with bevacizumab.

Additional highlights from the third quarter of 2023

• The FDA approved the first treatment indicated for patients with myelofibrosis and anemia: momelotinib (Ojjaara, GlaxoSmithKline), a JAK1/JAK2 and activin A receptor type 1 inhibitor
• 4 product indications were expanded with new approvals by the FDA
  • Bosutinib monohydrate (Bosulif^®; PF Prism CV) Philadelphia chromosome-positive chronic myeloid leukemia indication was expanded to include pediatric patients
  • Dabrafenib (Tafinlar^®; Novartis) tumor-agnostic indication expanded to include patients aged 1 and older
  • Trametinib (Mekinist^®; Novartis) tumor-agnostic indication now includes patients aged 1 and older
  • Luspatercept-aamt (Reblozyl^®; Bristol Myers Squibb) indication for anemia in patients with myelodysplastic syndrome expanded to no longer include “failing an erythropoiesis-stimulating agent”
• 5 product indications were expanded with new approvals by the EMA
  • Brentuximab vedotin (Adcetris^®; Takeda Pharmaceutical), now indicated for both stage III and stage IV Hodgkin lymphoma
  • Melphalan flufenamide (Pepaxti^®; Oncopeptides AB), now approved for third line or later (was previously approved for fourth line and beyond)
  • Nivolumab (Opdivo^®; Bristol Myers Squibb), for adjuvant treatment of stage IIB or IIC melanoma
  • Pembrolizumab (Keytruda^®; Merck Sharp & Dohme), for adjuvant treatment of NSCLC following chemotherapy and in combination with trastuzumab, fluoropyrimidine, and platinum-containing chemotherapy for first-line treatment of locally advanced unresectable or metastatic human epidermal growth factor receptor 2 (HER2)-positive gastric or gastroesophageal junction adenocarcinoma
  • Trastuzumab deruxtecan (Enhertu^®; Daiichi Sankyo), for treatment of NSCLC with HER2 mutation
• 1 product that previously received accelerated approval gained full approval by the FDA: avelumab (Bavencio^®; EMD Serono), for Merkel cell carcinoma
• Sacituzumab govitecan (Trodelvy^®; Gilead) also received marketing authorization from the EMA after a positive opinion in Q2 2023
• 1 new supportive care agent was approved by the FDA: fosaprepitant (Focinvez™; Spes Pharmaceuticals), for prevention of chemotherapy-induced nausea and vomiting
• 3 companion diagnostics were approved by the FDA
  • Therascreen PDGFRA kit, a PDGFRA assay approved by the FDA as a companion diagnostic for gastrointestinal tumors that may be eligible for treatment with avapritinib
  • LeukoStrat^® CDx FLT3 Mutation Assay, a companion diagnostic for quizartinib (see above)
  • FoundationOne CDx, a companion diagnostic for niraparib-abiraterone acetate (Akeega™; Janssen Biotech)
• 1 diagnostic received marketing authorization by the EMA: piflufolastat (18F) (Pylclari^®; Curium Pet France), for prostate cancer diagnosis, after receiving a positive opinion in Q2 2023
• A DNA blood test received de novo marketing authorization, a new regulatory classification from the FDA: Invitae’s Common Hereditary Cancers Panel, which identifies variants in 47 genes associated with an increased cancer risk
• Under its Project Orbis initiative, the FDA granted
  • Approval of dostarlimab-gxly (Jemperli; GlaxoSmithKline) in combination with chemotherapy for mismatch repair-deficient advanced or recurrent endometrial cancer (initially approved as monotherapy in 2021), in collaboration with the Australian Therapeutic Goods Administration (TGA), Health Canada, Switzerland’s Swissmedic, and United Kingdom’s Medicines and Healthcare products Regulatory Agency. Reviews are ongoing at the other regulatory agencies
  • Accelerated approval of elranatamab-bcmm (Elrexfio™; Pfizer) for relapsed/refractory multiple myeloma after at least 4 prior lines of therapy, in collaboration with the Australian TGA, the Brazilian Health Regulatory Agency, Health Canada, and Switzerland’s Swissmedic. Reviews are ongoing at the other agencies
  • Accelerated approval of talquetamab-tgvs (Talvey™; Janssen Biotech) for adults with relapsed or refractory multiple myeloma after at least 4 prior lines of therapy, in collaboration with the Australian TGA and Switzerland’s Swissmedic
• The EMA conditional marketing authorization for belantamab mafodotin (Blenrep; GlaxoSmithKline Ireland) in multiple myeloma was NOT RENEWED

The tables below summarize key Q3 2023 oncology approvals.