Lonapegsomatropin-tcgd (Skytrofa – Ascendis), a long-acting prodrug of recombinant human growth hormone (rhGH), has been approved by the FDA for once-weekly subcutaneous (SC) treatment of growth failure due to inadequate secretion of endogenous growth hormone in children ≥1 year old who weigh ≥11.5 kg. It is the first once-weekly rhGH product to be approved in the US; other available rhGH formulations are administered more frequently.

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 a 2% cream formulation of delgocitinib (Anzupgo – Leo), a Janus kinase (JAK) inhibitor, for treatment of moderate to severe chronic hand eczema (dermatitis) in adults who had an inadequate response to or are unable to use topical corticosteroids. Delgocitinib is the first drug to be approved in the US for this indication. Ruxolitinib, another JAK inhibitor, is available in a 1.5% cream formulation (Opzelura) for treatment of mild to moderate atopic dermatitis in patients ≥2 years old.

View the Comparison Table: Antiviral Drugs for Seasonal Influenza for 2025-2026

Anticoagulants are the drugs of choice for treatment and prevention of deep venous thrombosis (DVT) and pulmonary embolism (PE), collectively referred to as venous thromboembolism (VTE). US guidelines for treatment of VTE were updated in 2020 and 2021.

Human alpha1-proteinase inhibitor (Prolastin Cutter), possibly better known as alpha1-antitypsin (ATT), is now available in the USA for replacement therapy in patients with emphysema who have a congential deficiency of the protein.

(Ceredase - Genzyme) a modified form of the glycoprotein enzyme glucocerebrosidase prepared from human placenta, was recently approved by the US Food and Drug Administration as an 'orphan drug' for treatment of patients with the non-neurologic form of Gaucher's disease (Type 1).

Recent studies have caused concern about cardiomyopathy associated with doxorubicin (Adriamycin, and others), daunorubicin (Cerubidine, and others), idarubicin (Idamycin), mitoxantrone (Novantrone), and related anthracycline or anthraquinone anticancer drugs. Dexrazoxane (ADR-529, ICRF-187 - Adria), a piperazine derivative of ethylenediaminetetraacetic acid (EDTA), is now under investigation for prevention of this drug-induced cardiomyopathy.

The prostaglandin alprostadil injected into the corpus cavernosum (Caverject - Upjohn) can produce an erection in some men with erectile dysfunction (Medical Letter, 37:83, 1995). Now a pellet (microsuppository) formulation has become available for intraurethral administration of alprostadil (MUSE [Medicated Urethral System for Erection] - Vivus). It is marketed in a sterile foil pouch containing a pellet 1.4 mm in diameter and 3 or 6 mm long within the stem of a hollow applicator, which is inserted 3 cm deep into the urethra. Pressing a button pushes the pellet into the urethra.