The oral cyclin-dependent kinase (CDK) 4/6 inhibitor abemaciclib (Verzenio – Lilly), which was approved by the FDA in 2017 for treatment of hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative advanced or metastatic breast cancer, has now been approved for use in combination with endocrine therapy (tamoxifen or an aromatase inhibitor) for adjuvant treatment of patients with HR-positive, HER2-negative, node-positive, early breast cancer at high risk of recurrence and a Ki-67 score ≥20%.

Efgartigimod alfa-fcab (Vyvgart – Argenx), a first-in-class neonatal Fc receptor (FcRn) antagonist, has been approved by the FDA for IV treatment of generalized myasthenia gravis in adults who are anti-acetylcholine receptor (AChR) antibody positive. The IV complement inhibitor eculizumab (Soliris) was approved for the same indication in 2017.

The injectable glucagon-like peptide-1 (GLP-1) receptor agonist semaglutide (Wegovy), which was approved by the FDA in 2021 for chronic weight management in adults, has now been approved for chronic weight management in children ≥12 years old with an initial BMI in the ≥95th percentile for age and sex.Semaglutide is also available as Ozempic and Rybelsus for treatment of type 2 diabetes in adults.

The injectable glucagon-like peptide-1 (GLP-1) receptor agonist semaglutide (Wegovy) has been approved by the FDA to reduce the risk of major adverse cardiovascular events (MACE) in adults with established cardiovascular disease (CVD) and either obesity or overweight. Semaglutide is the first drug to be approved for cardiovascular risk reduction in this population. It is also approved in a lower-dose injectable formulation as Ozempic and in an oral formulation as Rybelsus (see Table 1).

The oral second-generation antipsychotic drug iloperidone (Fanapt – Vanda) has been approved by the FDA for acute treatment of manic or mixed episodes associated with bipolar I disorder in adults. First approved in 2009 for treatment of schizophrenia, iloperidone is the eighth second-generation antipsychotic to be approved for acute treatment of manic or mixed episodes of bipolar I disorder (see Table 1).

The FDA has approved ensartinib (Ensacove – Xcovery), an oral kinase inhibitor, for treatment of anaplastic lymphoma kinase (ALK)-positive, locally advanced or metastatic non-small cell lung cancer (NSCLC) in adults who have not previously received an ALK inhibitor (e.g, crizotinib [Xalkori], certinib [Zykadia], alectinib [Alecensa], brigatinib [Alunbrig], lorlatinib [Lorbrena]). All previously approved ALK inhibitors are also approved for first-line use.

The FDA has approved letermovir (Prevymis – Merck), a cytomegalovirus (CMV) DNA terminase complex inhibitor, for prophylaxis of CMV infection and disease in CMV-seropositive adult recipients of an allogeneic hematopoietic cell transplant (HCT). Letermovir is the first CMV DNA terminase complex inhibitor to be approved in the US and the only drug specifically indicated for CMV prophylaxis in HCT patients. It is not approved for treatment of CMV infection.

The FDA has approved two new drugs for sickle cell disease: crizanlizumab-tmca (Adakveo – Novartis), an IV P-selectin blocker, and voxelotor (Oxbryta – GBT), an oral hemoglobin S (HbS) polymerization inhibitor.

The FDA has approved mannitol powder for oral inhalation (Bronchitol – Chiesi) as add-on maintenance treatment to improve pulmonary function in adults with cystic fibrosis (CF). This product has been available in Europe for the same indication since 2012. Another formulation of mannitol inhalation powder (Aridol) is approved in the US for assessment of bronchial hyperresponsiveness.

The FDA has approved Lybalvi (Alkermes), a fixed-dose combination of the second-generation antipsychotic olanzapine (Zyprexa, and generics) and samidorphan, a new opioid antagonist, for treatment of adults with schizophrenia or with manic or mixed episodes of bipolar I disorder. The addition of samidorphan is intended to mitigate the weight gain that occurs with olanzapine. This is the first FDA approval for samidorphan.