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).

Antiplatelet drugs are the drugs of choice for prevention and treatment of arterial thrombosis. Anticoagulants are the drugs of choice for prevention and treatment of venous thromboembolism and for prevention of cardioembolic events in patients with atrial fibrillation.

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

The FDA has approved pegvisomant (Somavert Pfizer), a growth hormone receptor antagonist, for parenteral treatment of acromegaly in patients who are not candidates for or have had an inadequate response to surgery or other treatment. This review describes the disease of acromegaly and its standard treatment options. Also included are sections on the mechanism of action, pharmacokinetics, adverse effects, drug interactions, and the dosage and cost of pegvisomant. Results of clinical studies are discussed. The conclusion provides an overall assessment of the prospects for this new drug.

The FDA has approved pegaptanib sodium (Macugen - Pfizer/Eyetech) as an intravitreous injection for treatment of all subtypes of neovascular (wet) age-related macular degeneration (AMD). The only other approved treatment for this condition is photodynamic therapy with intravenous verteporfin (Visudyne), which is recommended only for patients with one subtype (predominantly classic) of neovascular AMD.