Shooting Cancer Highlights – Periodic Focus on regulatory status of new targeted therapies in oncology

EMA

• April 23 – Tislelizumab – NSCLC EGFR/ALK. The European Commission (EC) has approved the anti-PD-1 monoclonal antibody, across 3 indications for the first- and second-line treatment of adult patients with non–small cell lung cancer (NSCLC). In particular, tislelizumab, in combination with pemetrexed and platinum?containing chemotherapy, is indicated for the first-line treatment of adult patients with non-squamous non-small cell lung cancer whose tumours have PD-L1 expression on ?50% of tumour cells with no EGFR or ALK positive mutations. Moreover, tislelizumab as monotherapy is indicated for the treatment of adult patients with locally advanced or metastatic EGFR mutant or ALK positive NSCLC after prior platinum-based therapy. 

CHMP

• March 21 – Selpercatinib – Solid Tumours RET. The Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending a new indication for the RET receptor tyrosine kinase inhibitor: treatment, in monotherapy, of adults and adolescents with advanced RET fusion positive solid tumours, when treatment options not targeting RET provide limited clinical benefit, or have been exhausted.
• April 25 – Capivasertib – BC PIK3CA/AKT1/PTEN. The Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a marketing authorisation for the serine/threonine kinase inhibitor. Capivasertib is indicated in combination with fulvestrant for the treatment of adult patients with oestrogen receptor (ER)-positive, HER2-negative locally advanced or metastatic breast cancer with one or more PIK3CA/AKT1/PTEN-alterations following recurrence or progression on or after an endocrine-based regimen.
• April 25 – Alectinib – NSCLC ALK. The Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending extension of indications for the ALK tyrosine kinase inhibitor. Alectinib is recommended as adjuvant treatment, in monotherapy, following complete tumour resection for adult patients with ALK-positive non-small cell lung cancer (NSCLC) at high risk of recurrence.
• April 25 – Entrectinib – Solid Tumours NTRK. The Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending a change to the terms of the marketing authorisation for the TRK/ROS1/ALK inhibitor. Entrectinib, as monotherapy, is indicated for the treatment of adult and paediatric patients older than 1 month with solid tumours that have a NTRK gene fusion.
• April 25 – Amivantamab – NSCLC EGFR. The Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending a new indication for the EGF and MET receptor targeted antibody: first line treatment of adult patients with advanced non-small cell lung cancer (NSCLC) with activatingEGFR Exon 20 insertion mutations, in combination with carboplatin and pemetrexed. 

FDA

• March 1 – Amivantamab – NSCLC EGFR. The Food and Drug Administration (FDA) approved the EGF and MET receptor targeted antibody with carboplatin and pemetrexed for the first-line treatment of locally advanced or metastatic non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 20 insertion mutations, as detected by an FDA-approved test. The FDA also granted traditional approval to amivantamab for the treatment of adult patients with locally advanced or metastatic NSCLC with EGFR exon 20 insertion mutations, as detected by an FDA-approved test, whose disease has progressed on or after platinum-based chemotherapy.
• March 19 – Ponatinib – Ph+ ALL. The Food and Drug Administration (FDA) granted accelerated approval to the Bcr-Abl tyrosine kinase inhibitor with chemotherapy for the treatment of adult patients with newly diagnosed Philadelphia chromosome-positive acute lymphoblastic leukaemia (Ph+ ALL).
• March 21 – Fluorouracil injection products – DPD. The Food and Drug Administration (FDA) approved safety labelling changes regarding the risk of serious adverse reactions related to fluorouracil use in patients with dihydropyrimidine dehydrogenase (DPD) deficiency. Among revisions, a new subsection (Pharmacogenomics) has been added to section Clinical Pharmacology.
• April 18. Alectinib – NSCLC ALK. The Food and Drug Administration (FDA) approved the ALK tyrosine kinase inhibitor for adjuvant treatment following tumour resection in patients with anaplastic lymphoma kinase (ALK)-positive non-small cell lung cancer (NSCLC), as detected by an FDA-approved test.
• April 23 – Tovorafenib – LGG BRAF. The Food and Drug Administration (FDA) granted accelerated approval to the type II RAF kinase inhibitor for the treatment of patients 6 months of age and older with relapsed or refractory paediatric low-grade glioma (LGG) harbouring a BRAF fusion or rearrangement, or BRAF V600 mutation.
• March 26 – Revumenib – AML/ALL KMT2A. The Food and Drug Administration (FDA) has granted priority review to the first-in-class menin inhibitor for the treatment of adult and paediatric patients with relapsed or refractory (R/R) acute leukaemia harbouring KMT2A-rearrangements (KMT2Ar).
• April 7 – Sunvozertinib – NSCLC EGFR. The Food and Drug Administration (FDA) has granted a breakthrough therapy designation to the irreversible EGFR inhibitor targeting a wide spectrum of EGFR mutations with wild-type EGFR selectivity, as first -line treatment for patients with locally advanced or metastatic non–small cell lung cancer (NSCLC) harbouring EGFR exon 20 insertion (Exon20ins) mutations.
• April 9 – Tamibarotene – AML RARA. The Food and Drug Administration (FDA) has granted fast track designation to the first-in-class, selective RAR? agonist in combination with azacitidine and venetoclax for the treatment of patients with newly diagnosed acute myeloid leukaemia (AML) harbouring RARA gene overexpression, as detected by an FDA-approved test.
• April 22 – Ziftomenib – AML NPM1. The Food and Drug Administration (FDA) has granted a breakthrough therapy designation to the investigational drug targeting the menin-KMT2A/MLL protein-protein interaction for the treatment of heavily pretreated patients with relapsed/refractory (R/R) NPM1-mutant acute myeloid leukaemia (AML).