Shooting Cancer Highlights – Monthly focus on regulatory status of new targeted therapies in oncology – September and October 2023

EMA

• September 6 – Pembrolizumab – GC/GEJC HER2/PD-L1. The European Commission has extended the indication of pembrolizumab (anti-PD-1 antibody) to include treatment of locally advanced unresectable or metastatic HER2- positive gastric or gastro-oesophageal junction adenocarcinoma, in combination with trastuzumab, fluoropyrimidine and platinum containing chemotherapy, in adults whose tumours express PD-L1 with a CPS > 1.
• September 20 – Elacestrant – BC ER/HER2/ESR1. The European Commission has granted marketing authorisation to the estrogen receptor antagonist. Elacestrant monotherapy is indicated for the treatment of postmenopausal women, and men, with estrogen receptor (ER) positive, HER2-negative, locally advanced or metastatic breast cancer (BC) with an activating ESR1 mutation who have disease progression following at least one line of endocrine therapy including a CDK 4/6 inhibitor.
• September 22 – Tremelimumab  – NSCLC EGFR/ALK. The European Commission has extended the indication of tremelimumab (anti-CTLA-4 antibody) to include the first-line treatment of adults with metastatic non-small cell lung cancer (NSCLC) with no sensitising EGFR mutations or ALK positive mutations, in combination with durvalumab (anti-PD-L1 antibody) and platinum-based chemotherapy.

CHMP

• September 14 – Dabrafenib – LGG/HGG BRAF. The Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a marketing authorisation for the RAF kinase inhibitor, intended for the treatment of low- and high-grade glioma. Dabrafenib in combination with trametinib is indicated for the treatment of paediatric patients aged 1 year and older with low-grade glioma (LGG)    with a BRAF V600E mutation who require systemic therapy. Dabrafenib in combination with trametinib is indicated for the treatment of paediatric patients aged 1 year and older with high-grade glioma (HGG) with a BRAF V600E mutation who have received at least one prior radiation and/or chemotherapy treatment.

• September 14 – Quizartinib – AML FLT3-ITD. The Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a marketing authorisation for the fms-like tyrosine kinase 3 (FLT3) inhibitor. Quizartinib is indicated in combination with standard cytarabine and anthracycline induction and standard cytarabine consolidation chemotherapy, followed by maintenance monotherapy for adult patients with newly diagnosed acute myeloid leukaemia (AML) that is positive for FLT3 with internal tandem duplication (ITD).
• September 14 – Trastuzumab deruxtecan – NSCLC HER2. The Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending a new indication for the HER2-specific antibody-drug conjugate (ADC) to include treatment, as monotherapy, of adult patients with advanced non-small cell lung cancer whose tumours have an activating HER2 (ERBB2) mutation and who require systemic therapy following platinum-based chemotherapy with or without immunotherapy.
• October 12 – Dostarlimab – EC MMR/MSI. The Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending a new indication for the anti-PD-1 monoclonal antibody to include treatment of adult patients with mismatch repair deficient (dMMR)/microsatellite instability high (MSI H) primary advanced or recurrent endometrial cancer (EC) and who are candidates for systemic therapy, in combination with carboplatin and paclitaxel.
• October 12-  Pembrolizumab – GC/GEJC HER2/PD-L1. The Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending a new indication for the anti-PD-1 antibody. Pembrolizumab, in combination with fluoropyrimidine and platinum-containing chemotherapy, is indicated for the first-line treatment of locally advanced unresectable or metastatic HER2-negative gastric or gastro-oesophageal junction adenocarcinoma in adults whose tumours express PD-L1 with a CPS > 1.

FDA

• September 26 – Bosutinib – CML Ph. The Food and Drug Administration (FDA) approved the dual SRC and ABL tyrosine kinase inhibitor for paediatric patients 1 year of age and older with chronic phase (CP) Ph+ chronic myelogenous leukaemia (CML) that is newly diagnosed (ND) or resistant or intolerant (R/I) to prior therapy.
• October 11 – Encorafenib + Binimetinib – NSCLC BRAF. The Food and Drug Administration (FDA) approved encorafenib (BRAF-inhibitor) with binimetinib (MEK-inhibitor) for adult patients with metastatic non-small cell lung cancer (NSCLC) with a BRAF V600E mutation, as detected by an FDA-approved test.
• October 20 – Entrectinib – Solid Tumours NTRK. The Food and Drug Administration (FDA) granted accelerated approval to the tyrosine kinase inhibitor of NTRK1/2/3, ROS1 and ALK for paediatric patients older than 1 month with solid tumours that have a neurotrophic tyrosine receptor kinase (NTRK) gene fusion without a known acquired resistance mutation, are metastatic or where surgical resection is likely to result in severe morbidity, and have progressed following treatment or have no satisfactory standard therapy.
• October 24 – Ivosidenib – MDS IDH1. The Food and Drug Administration (FDA) approved the IDH1 inhibitor for adult patients with relapsed or refractory myelodysplastic syndromes (MDS) with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation, as detected by an FDA-approved test.
• September 12 – Tulmimetostat – EC ARID1A. The U.S. Food and Drug Administration (FDA) granted Fast Track designation for the dual inhibitor of EZH2 and EZH1, for the treatment of patients with advanced, recurrent or metastatic endometrial cancer (EC) harbouring AT-rich interacting domain containing protein 1A (ARID1A) mutations and who have progressed on at least one prior line of treatment.
• September 26 – IDE161 – OC BRCA 1/2. The U.S. Food and Drug Administration (FDA) has granted Fast Track designation to the selective inhibitor of poly (ADP-ribose) glycohydrolase (PARG) for the treatment of adult patients having advanced or metastatic ovarian cancer (OC) with germline or somatic BRCA 1/2 mutations, who are platinum resistant and have received prior antiangiogenic and poly (ADP-ribose) polymerase (PARP) inhibitor therapies.
• September 26 – MYTX-011 – NSCLC cMET. The U.S. Food and Drug Administration (FDA) has granted Fast Track designation to the investigational cMET-targeting antibody-drug conjugate (ADC), for the treatment of patients with non-small cell lung cancer (NSCLC) with cMET overexpression at any level, including low and intermediate.
• September 26 – ABM-1310 – GBM BRAF. The U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for the investigational BRAF inhibitor for the treatment of patients with Glioblastoma (GBM) carrying BRAF V600E
• September 28 – IDE161 – BC HR/HER2/BRCA 1/2. The U.S. Food and Drug Administration (FDA) has granted Fast Track designation to the investigational selective inhibitor of poly (ADP-ribose) glycohydrolase (PARG), for the treatment of adult patients having advanced or metastatic hormone receptor positive (HR+), Her2- breast cancer with germline or somatic BRCA 1/2 mutations who have progressed following treatment with at least one line of a hormonal therapy, a CDK4/6 inhibitor therapy and a poly (ADP-ribose) polymerase (PARP) inhibitor therapy.