Tarlatamab-dlle (Imdelltra – Amgen), a first-in-class bispecific delta-like ligand 3 (DLL3)-directed CD3 T-cell engager, has received accelerated approval from the FDA for treatment of extensive-stage small cell lung cancer (SCLC) in adults who had disease progression on or after platinum-based chemotherapy. It is the first bispecific DLL3-directed CD3 T-cell engager to be approved in the US for this indication. Most patients with SCLC have a response to initial treatment, but progression generally occurs within a few months and overall survival is usually less than 8 months....

Marstacimab-hncq (Hympavzi – Pfizer), a tissue factor pathway inhibitor (TFPI) antagonist, has been approved by the FDA for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in patients ≥12 years old who have hemophilia A without factor VIII inhibitors or hemophilia B without factor IX inhibitors. It is the first hemophilia treatment to be approved in the US that targets an inhibitor of coagulation and the first to become available in prefilled pens and syringes for subcutaneous administration.

The FDA has approved daridorexant (Quviviq – Idorsia), an orexin receptor antagonist, for treatment of sleep-onset and/or sleep-maintenance insomnia in adults. Daridorexant is the third orexin receptor antagonist to be approved for this indication; suvorexant (Belsomra) and lemborexant (Dayvigo) were approved earlier.

Caplacizumab-yhdp (Cablivi – Sanofi /Genzyme), a von Willebrand factor-directed antibody fragment, has been approved by the FDA for use in combination with plasma exchange and immunosuppressive therapy for treatment of immune-mediated thrombotic thrombocytopenic purpura (iTTP; formerly called acquired thrombotic thrombocytopenic purpura [aTTP]) in adults. It is the first drug to be approved in the US for this indication.

The FDA has approved anifrolumab-fnia (Saphnelo – AstraZeneca), a type I interferon receptor antagonist, for IV treatment of adults with moderate to severe systemic lupus erythematosus (SLE) who are receiving standard treatment. Anifrolumab has not been studied in patients with severe active lupus nephritis or severe active CNS lupus. It is the first type I interferon receptor antagonist to become available in the US.

Mobocertinib (Exkivity – Takeda), an oral kinase inhibitor, has received accelerated approval from the FDA for treatment of locally advanced or metastatic non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 20 insertion mutations in adults whose disease has progressed on or after platinum-based chemotherapy. Accelerated approval was based on the overall response rate and duration of response. Mobocertinib is the second drug to become available in the US for this indication; the IV EGFR-MET bispecific antibody amivantamab (Rybrevant) was approved...

Lecanemab-irmb (Leqembi – Biogen/Eisai), an IV amyloid beta-directed monoclonal antibody, has received accelerated approval from the FDA for treatment of Alzheimer's disease. The label states that treatment with Leqembi should be initiated in patients with mild cognitive impairment (MCI) or mild dementia, the population in which the drug was initiated in clinical trials. Lecanemab is the second IV amyloid-beta directed monoclonal antibody to be approved for this indication; aducanumab (Aduhelm) was approved in 2021 and has the same restrictions on treatment...

The oral kinase inhibitors dabrafenib (Tafinlar – GSK) and trametinib (Mekinist – Novartis) have received accelerated approval by the FDA for use together for a fifth indication: treatment of unresectable or metastatic solid tumors with a BRAF V600E mutation in patients ≥6 years old who have progressed following prior treatment and have no satisfactory alternative treatment options. The combination is not approved for treatment of colorectal cancer because of known intrinsic resistance to BRAF inhibition and dabrafenib is not approved for use in patients with wild-type BRAF...

The oral kinase inhibitor capivasertib (Truqap – AstraZeneca), a first-in-class AKT inhibitor, has been approved by the FDA for use in combination with the selective estrogen receptor degrader (SERD) fulvestrant (Faslodex, and generics) for treatment of hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative, locally advanced or metastatic breast cancer in adults with one or more PIK3CA/AKT1/PTEN-alterations who had disease progression on at least one endocrine-based regimen for metastatic disease or recurrence on or within 12 months of completing...

Trofinetide (Daybue – Acadia), a synthetic analog of glycine-proline-glutamate, has been approved by the FDA for treatment of Rett syndrome in patients ≥2 years old. It is the first drug to be approved in the US for treatment of Rett syndrome.