The oral kinase inhibitor osimertinib (Tagrisso – AstraZeneca), which has been available for years for treatment of non-small cell lung cancer (NSCLC) in adults with epidermal growth factor receptor (EGFR) exon 19 deletions or exon 21 L858R mutations, has now been approved for treatment of unresectable stage III EGFR-mutated NSCLC. About 20-30% of patients with NSCLC have locally advanced stage III NSCLC, and 60-90% of these patients have unresectable disease. Osimertinib is the first targeted therapy to be approved for the new indication.

Isatuximab-irfc (Sarclisa – Sanofi), a CD38-directed cytolytic antibody, has been approved by the FDA for treatment of newly diagnosed multiple myeloma in adults who are not eligible for autologous stem cell transplantation (ASCT). The drug was approved earlier for treatment of relapsed or treatment-refractory multiple myeloma.

Zolbetuximab-clzb (Vyloy – Astellas), a claudin 18.2 (CLDN18.2)-directed cytolytic antibody, has been approved by the FDA for use in combination with fluoropyrimidine- and platinum-containing chemotherapy for first-line treatment of locally advanced unresectable or metastatic human epidermal growth factor receptor 2 (HER2)-negative gastric or gastroesophageal junction (GEJ) adenocarcinoma in patients who have CLDN18.2-positive tumors. It is the first monoclonal antibody to be approved in the US that selectively targets CLDN18.2.

Vimseltinib (Romvimza – Deciphera), an oral kinase inhibitor, has been approved by the FDA for treatment of symptomatic tenosynovial giant cell tumor in adults for whom surgical resection would cause worsening functional limitation or severe morbidity. The kinase inhibitor pexidartinib (Turalio) was approved for a similar indication in 2019.

Telisotuzumab vedotin-tllv (Emrelis – Abbvie), a c-Met-directed antibody and microtubule inhibitor conjugate, has received accelerated approval from the FDA for treatment of locally advanced or metastatic nonsquamous non-small cell lung cancer (NSCLC) in adults with high c-Met protein overexpression [≥50% of tumor cells with strong (3+) staining] who received prior systemic therapy. Accelerated approval was based on the overall response rate and duration of response.

Dordaviprone (Modeyso – Jazz), an oral protease activator, has received accelerated approval from the FDA for treatment of diffuse midline gliomas harboring a H3 K27M mutation in patients ≥ year old who had disease progression following prior treatment. It is the first drug to be approved in the US for this indication.

The FDA has approved the interleukin-2 (IL-2) receptor-directed fusion protein denileukin diftitox (Lymphir – Citius) for treatment of relapsed or refractory stage I-III cutaneous T-cell lymphoma in adults who received at least one prior systemic therapy. Lymphir is a reformulated, purified version of Ontak, which was voluntarily withdrawn from the market in 2014 because of manufacturing issues.

The FDA has approved Vybrique (IBSA), an oral film formulation of the phosphodiesterase type 5 (PDE5) inhibitor sildenafil, for treatment of erectile dysfunction (ED). Sildenafil oral tablets (Viagra, and generics) have been available since 1998. The manufacturer is promoting the new product as a "discreet, on-the-go" option that can be taken without water.

Plozasiran (Redemplo – Arrowhead), an apolipoprotein C-III (apoC-III)-directed small interfering ribonucleic acid (siRNA), has been approved by the FDA to reduce triglyceride levels in adults with familial chylomicronemia syndrome (FCS). It is the second drug to be approved in the US for this indication; the apoC-III-directed antisense oligonucleotide olezarsen (Tryngolza) was approved in 2024.

Solifenacin succinate (VESIcare - Yamanouchi and GlaxoSmithKline) and darifenacin hydrobromide (Enablex - Novartis) have been approved by the FDA for oral treatment of overactive bladder.