The FDA has approved use of oral leucovorin calcium (folinic acid) for treatment of cerebral folate transport deficiency in patients who have a confirmed variant in the folate receptor 1 gene (FOLR1-CFTD). It is the first drug to be approved for this extremely rare disorder.

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...

The FDA has approved the oral tyrosine kinase inhibitor repotrectinib (Augtyro – BMS) for treatment of locally advanced or metastatic ROS1-positive non-small cell lung cancer (NSCLC) in adults. Repotrectinib is the third oral tyrosine kinase inhibitor to be approved for this indication in the US; crizotinib (Xalkori) and entrectinib (Rozlytrek) were approved earlier.

The FDA has approved the nonsteroidal mineralocorticoid receptor antagonist (MRA) finerenone (Kerendia) to reduce the risk of cardiovascular death, hospitalization for heart failure (HF), and urgent HF visits in adults with HF with a left ventricular ejection fraction (LVEF) ≥40%. Finerenone was approved in 2021 to reduce the risk of kidney disease progression and cardiovascular events in adults with chronic kidney disease (CKD) associated with type 2 diabetes.

The sodium-glucose cotransporter 2 (SGLT2) inhibitor empagliflozin (Jardiance – Boehringer Ingelheim) has been approved by the FDA for a third indication: to reduce the risk of hospitalization for heart failure (HF) and cardiovascular death in patients with heart failure with reduced ejection fraction (HFrEF), with or without type 2 diabetes. The SGLT2 inhibitor dapagliflozin (Farxiga) was approved for the same indication in 2020. Empagliflozin has been granted breakthrough therapy designation for treatment of patients with heart failure with preserved ejection fraction...

On November 19, the FDA expanded the Emergency Use Authorizations (EUAs) for the mRNA-based COVID-19 vaccines manufactured by Pfizer/BioNTech (Comirnaty) and Moderna (Spikevax) to include administration of a booster dose for all adults ≥18 years old after primary immunization with either the same COVID-19 vaccine or a different one. Booster doses of these vaccines were previously authorized only for select populations (age ≥65 years or persons at high risk for severe COVID-19). The EUA for the adenovirus-based vaccine manufactured by Johnson & Johnson was amended in October 2021...

Invega Hafyera (Janssen), a long-acting, extended-release (ER) formulation of the second-generation antipsychotic paliperidone palmitate, has been approved by the FDA for twice-yearly IM treatment of schizophrenia in adults who have been adequately treated with another injectable ER formulation of the drug (Invega Sustenna or Invega Trinza). It is the first drug to become available in the US for twice-yearly treatment of schizophrenia.

The oral kinase inhibitor futibatinib (Lytgobi – Taiho) has received accelerated approval from the FDA for treatment of adults with previously treated, unresectable, locally advanced or metastatic intrahepatic cholangiocarcinoma with fibroblast growth factor receptor 2 (FGFR2) fusions or other rearrangements. Accelerated approval was based on the overall response rate and duration of response. Futibatinib is the second drug to be approved for this indication; pemigatinib (Pemazyre) was approved earlier.

The investigational anti-complement component 5a (C5a) antibody vilobelimab (Gohibic – InflaRx) has been granted an FDA Emergency Use Authorization (EUA) for IV treatment of hospitalized adults with COVID-19 beginning within 48 hours after invasive mechanical ventilation (IMV) or extracorporeal membrane oxygenation (ECMO) is started. Vilobelimab is the first anti-C5a antibody to become available in the US.

The FDA has approved Inlexzo, a gemcitabine intravesical system, for treatment of Bacillus Calmette-Guérin (BCG)-unresponsive nonmuscle invasive bladder cancer (NMIBC) with carcinoma in situ, with or without papillary tumors. The Inlexzo system is placed into the bladder to deliver gemcitabine for 3 weeks.