EMA
October 28. Selumetinib – PN NF1. The European Commission (EC) has approved the oral, selective MEK inhibitor for the treatment of symptomatic, inoperable plexiform neurofibromas (PN) in adult patients with neurofibromatosis type 1 (NF1). NF1 is a rare, progressive, genetic condition that is caused by a spontaneous or inherited mutation in the NF1 Up to 50% of people living with NF1 may develop a type of non-malignant tumour called PN that may affect the brain, spinal cord and nerves.
CHMP
October 16. Sugemalimab – NSCLC EGFR/ALK/ROS1. The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion, recommending a new indication for the anti-PD-L1 monoclonal antibody. Sugemalimab, as monotherapy, is indicated for the treatment of unresectable stage III non-small cell lung cancer (NSCLC) with no sensitising EGFR mutations, or ALK, ROS1 genomic tumour aberrations in adults whose tumours express PD-L1 on ≥1% of tumour cells and whose disease has not progressed following platinum-based chemoradiotherapy.
October 16. Asciminib – CML BCR-ABL. The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion and recommended granting marketing authorisation for the tyrosine kinase inhibitor (TKI) of ABL/BCR-ABL1 for the treatment of adult patients with Philadelphia chromosome-positive chronic myeloid leukaemia in chronic phase (Ph+ CML-CP) in all lines of treatment, not only after treatment with two or more TKIs. If approved, asciminib will be indicated for adults with CML, both newly diagnosed and previously treated, expanding access to four times as many patients in Europe.
FDA
October 24. Revumenib – AML NPM1. The U.S. Food and Drug Administration (FDA) has approved the menin inhibitor for the treatment of relapsed or refractory (R/R) acute myeloid leukaemia (AML) with a susceptible nucleophosmin 1 (NPM1) mutation in adult and paediatric patients one year and older who have no satisfactory alternative treatment options. Revumenib previously received FDA approval in 2024 for the treatment of R/R acute leukaemia with a KMT2A translocation in adult and paediatric patients one year and older.
October 1. ETX-636 – BC PIK3CA. The U.S. Food and Drug Administration (FDA) granted Fast Track Designation to the pan mutant-specific allosteric PI3Kα inhibitor and degrader for the treatment of adult patients with PIK3CA-mutant, hormone receptor positive (HR+)/human epidermal growth factor negative (HER2-) advanced breast cancer.
October 23. Zenocutuzumab – CCA NRG1. The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to the HER2xHER3 bispecific antibody for the treatment of adults with advanced unresectable or metastatic cholangiocarcinoma (CCA) harbouring a neuregulin 1 (NRG1) gene fusion.
October 27. Daraxonrasib – PDCA RAS. The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to the RAS(ON) multi-selective inhibitor for the treatment of pancreatic ductal adenocarcinoma (PDCA) harbouring RAS mutations.
AIFA
October 13. Inavolisib – BC PIK3CA. The Italian Medicines Agency (AIFA) has approved the PI3Kα inhibitor, in combination with palbociclib and fulvestrant, for the treatment of adult patients with PIK3CA-mutated, oestrogen receptor (ER)-positive, HER2-negative, locally advanced or metastatic breast cancer following recurrence during or within 12 months of completion of adjuvant endocrine therapy.
October 21. Erdafitinib – UC FGFR3. The Italian Medicines Agency (AIFA) has approved the reimbursement of the fibroblast growth factor receptor tyrosine kinase inhibitor as monotherapy for the treatment of adult patients with unresectable or metastatic urothelial carcinoma (UC), harbouring susceptible genetic FGFR alterations who have previously received at least one line of therapy with a PD-1 or PD-L1 inhibitor for unresectable or metastatic disease.