Proton pump inhibitors (PPIs), which are used for treatment of gastroesophageal reflux disease (GERD) and for prevention of upper gastrointestinal adverse effects caused by NSAIDs and aspirin, are one of the most commonly prescribed classes of drugs in the US. All PPIs are similarly effective and generally well tolerated, but their long-term use has been associated with a number of safety concerns. Recommendations addressing these concerns have recently been published.

The FDA has approved deutetrabenazine (Austedo – Teva), a vesicular monoamine transporter 2 (VMAT2) inhibitor, for treatment of chorea associated with Huntington's disease and, more recently, for treatment of tardive dyskinesia in adults. It is the second VMAT2 inhibitor to be approved for each of these indications; tetrabenazine (Xenazine, and generics) was approved earlier for Huntington's chorea and valbenazine (Ingrezza) was recently approved for treatment of adults with tardive dyskinesia.

The FDA has approved baloxavir marboxil (Xofluza – Shionogi/Genentech), the first polymerase acidic (PA) endonuclease inhibitor, for single-dose, oral treatment of acute uncomplicated influenza in patients ≥12 years old. Baloxavir is the first drug with a new mechanism of action to be approved for treatment of influenza in almost 20 years.

The FDA has approved crizotinib (Xalkori – Pfizer), an oral tyrosine kinase inhibitor, for treatment of locally advanced or metastatic non-small cell lung cancer (NSCLC) with the anaplastic lymphoma kinase (ALK) translocation, which is found in about 4-5% of lung cancers. A diagnostic test (Vysis ALK Break Apart FISH Probe Kit – Abbott Molecular) is available to detect translocations of the ALK gene in tumor samples; these translocations occur predominantly in nonsmokers with adenocarcinoma.

Long-term use of bisphosphonates for prevention and treatment of osteoporosis has been associated with osteonecrosis of the jaw, atypical fractures of the femur, and possibly esophageal cancer. So for how long should patients take them?

The FDA has approved the MiniMed 530G (Medtronic), an insulin pump used in combination with a continuous glucose monitoring sensor (Enlite) that can stop delivery of insulin when interstitial glucose reaches a prespecified low level. It is the only sensoraugmented insulin pump available in the US with this capability.

Brexucabtagene autoleucel (Tecartus – Kite) has been approved by the FDA for treatment of adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (ALL). It was previously approved for treatment of relapsed or refractory mantle cell lymphoma. Tecartus is an individualized cellular product prepared from the patients own T cells, which are genetically modified to express chimeric antigen receptors (CAR) and then infused back into the patient. The CAR T-cell immunotherapy tisagenlecleucel (Kymriah) was approved in 2017 for treatment of relapsed or refractory...

Exagamglogene autotemcel (Casgevy – Vertex), a cell-based gene therapy recently approved for treatment of sickle cell disease1, has now been approved by the FDA for treatment of patients ≥12 years old with transfusion-dependent beta thalassemia. Casgevy is the first gene therapy that uses CRISPR/Cas9 gene-editing technology to be approved in the US for any disorder. Betibeglogene autotemcel (Zynteglo), an autologous lentiviral vector cell-based gene therapy, was approved in the US in 2022 for treatment of transfusion-dependent beta thalassemia.

The CDC has issued a recommendation for use of a single dose of doxycycline for post-exposure prophylaxis (PEP) of bacterial sexually transmitted infections (STIs), specifically syphilis, chlamydia, and gonorrhea, in men who have sex with men (MSM) and transgender women (TGW) who had a bacterial STI within the previous 12 months.

Revakinagene taroretcel-lwey (Encelto – Neurotech), an intravitreal allogeneic cell-based gene therapy, has been approved by the FDA for treatment of idiopathic macular telangiectasia type 2 in adults. It is the first treatment to be approved in the US for this indication.