The FDA has approved vutrisiran (Amvuttra – Alnylam), a subcutaneously injected small interfering RNA (siRNA), to reduce cardiovascular hospitalizations, urgent heart failure visits, and cardiovascular death in adults with wild-type or variant transthyretin amyloid cardiomyopathy (ATTR-CM). Vutrisiran is the first siRNA to be approved in the US for this indication; it was approved earlier for treatment of polyneuropathy associated with hereditary transthyretin-mediated amyloidosis.

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.

Consumption of alcohol has increased during the COVID-19 pandemic. The Diagnostic and Statistical Manual for Mental Disorders, 5th edition (DSM-5) defines alcohol use disorder (AUD; previously called alcohol dependence) as meeting ≥2 of the 11 criteria listed in Table 1 in the past year. The lifetime prevalence of AUD in the US population has been estimated to be about 30%. Despite this high prevalence and the associated morbidity, mortality, and costs, only 3 drugs are FDA-approved for treatment of the disorder.

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.

Maribavir (Livtencity – Takeda), an oral cytomegalovirus (CMV) pUL97 kinase inhibitor, has been approved by the FDA for treatment of post-transplant CMV infection refractory to standard antiviral therapy in patients ≥12 years old who weigh at least 35 kg.

The FDA has approved ciltacabtagene autoleucel (Carvykti – Janssen), a B-cell maturation antigen (BCMA)-directed genetically-modified cellular product, for treatment of relapsed or refractory multiple myeloma in adults who received ≥4 prior lines of therapy, including a proteasome inhibitor, an immunomodulatory drug, and an anti-CD38 monoclonal antibody. Carvykti is an individualized cellular product prepared from the patient's own T cells, which are genetically modified to express chimeric antigen receptors (CAR) and then infused back into the patient. Idecabtagene...

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...

Opdualag (BMS), a fixed-dose combination of two immune checkpoint inhibitors — nivolumab (Opdivo), a programmed death receptor-1 (PD-1) inhibitor, and relatlimab-rmbw, a lymphocyte-activation gene-3 (LAG-3) blocking antibody — has been approved by the FDA for treatment of unresectable or metastatic melanoma in patients ≥12 years old. Relatlimab, which is only available in combination with nivolumab, is the first LAG-3 blocking antibody to become available in the US. Immune checkpoint inhibitors, including the anti-CTLA-4 antibody ipilimumab (Yervoy) and the PD-1 inhibitors...

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 selpercatinib (Retevmo – Lilly) and pralsetinib (Gavreto – Blueprint Medicines/Genentech) have been approved by the FDA for treatment of advanced or metastatic RET-mutant or RET fusion-positive cancers. They are the first drugs to be approved specifically for certain RET-driven cancers. Both drugs were granted accelerated approval based on overall response rates and the duration of response.