The FDA has approved inclisiran (Leqvio – Novartis), a small interfering RNA (siRNA) directed to proprotein convertase subtilisin/kexin type 9 (PCSK9) mRNA, as an adjunct to diet and maximally tolerated statin therapy for subcutaneous (SC) treatment of adults with heterozygous familial hypercholesterolemia (HeFH) or clinical atherosclerotic cardiovascular disease (ASCVD) who require additional lowering of low-density lipoprotein cholesterol (LDL-C). Inclisiran is the first FDA-approved PCSK9-directed siRNA therapeutic agent.

The FDA has approved a sublingual film formulation of the alpha-2 adrenergic receptor agonist dexmedetomidine (Igalmi — BioXcel Therapeutics) for acute treatment of agitation associated with schizophrenia or bipolar I or II disorder in adults. It is the first alpha-2 agonist and the only sublingual film to be approved for this indication. Dexmedetomidine has been available for many years in an injectable formulation (Precedex) for sedation in the intensive care unit and to facilitate procedures such as mechanical ventilation.

The FDA has approved the recombinant chimeric anti-CD20 antibody ublituximab-xiiy (Briumvi – TG Therapeutics) for IV treatment of adults with relapsing forms of multiple sclerosis (MS), including clinically isolated syndrome (initial neurological episode), relapsing-remitting disease, and active secondary progressive MS (SPMS). Two other anti-CD20 antibodies, IV ocrelizumab (Ocrevus) and SC ofatumumab (Kesimpta), are also FDA-approved for these indications.

Seladelpar (Livdelzi – Gilead), a peroxisome proliferator-activated receptor (PPAR)-delta agonist, has received accelerated approval from the FDA for use in combination with ursodeoxycholic acid (ursodiol, UDCA; Urso Forte, and generics) for treatment of primary biliary cholangitis (PBC) in adults who had an inadequate response to UDCA and as monotherapy in those unable to tolerate UDCA. Accelerated approval was based on a reduction in alkaline phosphatase (ALP) levels. Seladelpar is the second PPAR agonist to be approved in the US for this indication; elafibranor (Iqirvo)...

The FDA has approved pivmecillinam (Pivya – Utility Therapeutics), an oral penicillin-class antibacterial drug, for treatment of uncomplicated urinary tract infections (uUTIs) caused by susceptible isolates of Escherichia coli, Proteus mirabilis, or Staphylococcus saprophyticus in adult females. Pivmecillinam was approved by the FDA in 2024 but only recently became available in the US. It has been used in Europe for over 40 years.

The FDA has approved the CD19-directed cytolytic antibody inebilizumab (Uplizna – Amgen) for IV treatment of generalized myasthenia gravis (gMG) in adults with anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibodies. The neonatal Fc receptor (FcRn) blockers rozanolixizumab (Rystiggo), nipocalimab (Imaavy), and efgartigimod alfa (Vyvgart; Vyvgart Hytrulo) are also approved for such use (see Table 1).

Andexanet alfa (Andexxa – AstraZeneca), which received accelerated approval from the FDA in 2018 for urgent reversal of the anticoagulant effect of the direct factor Xa inhibitors apixaban (Eliquis) and rivaroxaban (Xarelto), has voluntarily been withdrawn from the market. Andexxa was the only drug FDA-approved for this indication in the US.

Gastroesophageal reflux disease (GERD) is the most common GI condition encountered in the outpatient setting; it affects about 20% of people in the US.

Irritable bowel syndrome (IBS) is a common disorder characterized by recurrent abdominal pain and altered bowel habits, often accompanied by bloating. IBS is classified by its predominant bowel symptom: constipation (IBS-C), diarrhea (IBS-D), mixed type (IBS-M), or unclassified (IBS-U). Since the exact cause of IBS is unknown, the goal of treatment is symptom control. Some over-the-counter (OTC) products and prescription drugs for IBS are listed in Tables 1-4. The safety of these drugs during pregnancy and lactation is described in Table 5 (online only).

Breztri Aerosphere (AstraZeneca), a three-drug inhaler containing the inhaled corticosteroid (ICS) budesonide, the long-acting antimuscarinic agent (LAMA) glycopyrrolate, and the long-acting beta2-agonist (LABA) formoterol fumarate, has been approved by the FDA for maintenance treatment of asthma in patients ≥12 years old. It was approved in 2020 for treatment of chronic obstructive pulmonary disease (COPD). Breztri Aerosphere is the second ICS/LAMA/LABA combination to become available for treatment of asthma. Trelegy Ellipta, which contains the ICS fluticasone, the...