Novartis announced that the FDA approved dabrafenib (Tafinlar) with trametinib (Mekinist) for children age 1 and older with low-grade glioma with a BRAF^V600E mutation. The agency also OK’d new oral formulations of both drugs for patients who cannot swallow pills. This is the first approval of a systemic therapy as an initial treatment for children with this condition.

Drugmakers that raised prices faster than the rate of inflation on 27 medicines will be fined, the Biden administration announced, and will have to pay back the difference to Medicare. Six of the drugs treat blood cancers: axicabtagene ciloleucel (Yescarta; Gilead), brexucabtagene autoleucel (Tecartus; Kite/Gilead), tagraxofusp (Elzonris; Menarini Group), pralatrexate (Folotyn; Acrotech Biopharma), sargramostim (Leukine; Partner Therapeutics), and pentostatin (Nipent; Pfizer). The lung cancer drug amivantamab (Rybrevant; Johnson & Johnson) and the bladder cancer drug enfortumab vedotin (Padcev; Seagen), as well as Helsinn Healthcare’s netupitant/palonosetron (Akynzeo), also made the list. Medicare beneficiaries will soon see prices of these drugs reduced accordingly.

At the annual meeting of the European Association of Urology in Milan, Italy, researchers reported that prostatectomy and radiotherapy cut the incidence of prostate cancer metastasis, local progression, and long-term use of androgen deprivation therapy in half compared with active monitoring (N Engl J Med 2023 Mar 11 [Epub ahead of print]). However, these reductions had no impact on survival after 15 years, but radical treatment was linked to urinary and bowel problems and sexual dysfunction. The data come from the ProtecT study, which enrolled 1,643 men in the UK with localized prostate cancer. Data on patients’ quality of life and physical functioning were also reported (NEJM Evid 2023 Mar 11 [Epub ahead of print]).

Researchers reported that, in patients with relapsed/refractory multiple myeloma who had already received two to four therapies, Bristol Myers Squibb’s idecabtagene vicleucel (ide-cel; Abecma) significantly improved progression-free survival (PFS) compared with standard regimens (N Engl J Med 2023;388:1002–14). After a median follow-up of 18.6 months, patients who received the BCMA-directed chimeric antigen receptor T-cell therapy had a median PFS of 13.3 months compared with 4.4 months for those who received a standard drug regimen. The percentage of patients who experienced a complete response or a stringent complete response was also higher with ide-cel compared with standard regimens—39% vs. 5%, respectively.

Clinical trial data show that immune checkpoint blockade can prompt lymph nodes to produce cancer-fighting T cells, suggesting that leaving lymph nodes near a tumor intact could boost the drugs’ effectiveness (Cell 2023;186:1127–43). Instead of having immediate surgery to remove the tumor and lymph nodes, the trial’s 12 patients, all of whom had head and neck cancers that hadn’t metastasized beyond the lymph nodes, received one cycle of the PD-L1 inhibitor atezolizumab (Tecentriq; Genentech) prior to surgery. Examining tissue samples from before and after surgery, the researchers found greater T-cell activation in the nodes and more immune cells in the blood.