New Treatment Options in Oncology: FDA and EMA Drug Approvals in Q1 2023 – Focus on Solid Tumors

In the first quarter of 2023, the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) approved more than 25 new oncology agents and new indications for previously approved agents. The majority of these approvals were treatments for skin, prostate, breast, and lung cancers. There were 9 biologics and 5 programmed cell death protein 1 (PD-1) inhibitors approved. A key highlight was the FDA approvals of dabrafenib mesylate (Tafinlar^®; Novartis) and trametinib (Mekinist^®; Novartis) pediatric indication and new oral solutions, which were also granted priority review, breakthrough designation, and orphan drug designation. This was the first systemic therapy approval for first-line treatment of pediatric patients with low-grade glioma with a BRAF V600E mutation. Another approval of interest was that of pembrolizumab (Keytruda^®; Merck Sharp & Dohme) for unresectable or metastatic microsatellite instability-high or mismatch repair-deficient solid tumors, the first approval for an immunotherapy on the basis of a predictive biomarker, regardless of solid tumor type.

Additional highlights from the first quarter of 2023 include

• Elacestrant (Orserdu^®; Stemline Therapeutics), an estrogen receptor antagonist, received approval by the FDA with priority review and fast track designation for treatment of estrogen receptor-positive, human epidermal growth factor receptor 2 (HER2)-negative, ESR1-mutated advanced or metastatic breast cancer with disease progression following at least 1 line of endocrine therapy
  • A companion diagnostic, Guardant360 CDx assay, was also approved
• Two ivosidenib formulations, Tibsovo^® and Tidhesco^®, both with an orphan medicine designation and manufactured by Les Laboratoires Servier, received a positive opinion by the EMA for treatment of acute myeloid leukemia
• There were several new indications approved for existing therapies
  • Abemaciclib (Verzenio^®; Eli Lilly and Company) by the FDA, in combination with an aromatase inhibitor as initial endocrine-based therapy for advanced or metastatic breast cancer and in combination with endocrine therapy for adjuvant treatment of early breast cancer
  • Cemiplimab (Libtayo^®; Regeneron) by the EMA, in combination with platinum-based chemotherapy for treatment of PD-1 ligand 1 (PD-L1)–positive non-small cell lung cancer (NSCLC)
  • Darolutamide (Nubeqa^®; Bayer AG) by the EMA for metastatic hormone-sensitive prostate cancer, in combination with docetaxel and androgen deprivation therapy
  • Lisocabtagene maraleucel (Breyanzi^®; Bristol-Myers Squibb) by the EMA for diffuse large B-cell lymphoma, high-grade B-cell lymphoma, primary mediastinal large B-cell lymphoma, and follicular lymphoma stage 3B that relapsed within 12 months from completion of or is refractory to chemoimmunotherapy
  • Zanubrutinib (Brukinsa^®; BeiGene) by the FDA for chronic or small lymphocytic lymphoma, with orphan drug designation
• Three product indications were expanded
  • Ipilimumab (Yervoy^®; Bristol-Myers Squibb) melanoma indication was revised to include nivolumab (Opdivo^®; Bristol-Myers Squibb)
  • Nivolumab melanoma indications were expanded to include pediatric patients aged 12 and older
  • Leuprolide acetate (Lutrate^® Depot Kit; InvaGen Pharmaceuticals) indication was expanded with the removal of “palliative”
• One new diagnostic was approved for prostate cancer diagnosis
  • Gallium Ga 68 gozetotide (Illuccix^®; Telix) approved by the FDA for selection of patients with metastatic prostate cancer, for whom lutetium Lu 177 vipivotide tetraxetan (Pluvicto^®; Novartis) prostate-specific membrane antigen–directed therapy is indicated
• Under its Project Orbis initiative, which provides a framework for concurrent submission and review of oncology drugs among international partners, the FDA granted
• Full approval (previously accelerated approval) for dostarlimab-gxly (Jemperli^®; GlaxoSmithKline) to treat endometrial cancer, in collaboration with Australia’s Therapeutic Goods Administration, Health Canada, and Switzerland’s Swissmedic. Reviews are ongoing at the other regulatory agencies
• New NSCLC indication approval for pembrolizumab (Keytruda; Merck Sharp & Dohme), also in collaboration with Australia’s Therapeutic Goods Administration, Health Canada, and Switzerland’s Swissmedic
  • Approved as single-agent, adjuvant treatment following resection and platinum-based chemotherapy for adults with stage IB (T2a ≥4 cm), II, or IIIA NSCLC
• New indication for tucatinib (Tukysa^®; Seagen) in combination with trastuzumab for RAS wild-type, HER2-positive, unresectable or metastatic colorectal cancer that has progressed following chemotherapy (fluoropyrimidine, oxaliplatin, and irinotecan based). Review was conducted in collaboration with (and is ongoing at) Australia’s Therapeutic Goods Administration

The tables below summarize key Q1 oncology approvals:

Sources:

https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm?event=reportsSearch.process

https://www.fda.gov/drugs/resources-information-approved-drugs/oncology-cancer-hematologic-malignancies-approval-notifications

https://www.ema.europa.eu/en/committees/chmp/chmp-agendas-minutes-highlights