The FDA has approved Onyda XR (Tris), an extended-release (ER) oral suspension formulation of the alpha_2-adrenergic agonist clonidine, for use as monotherapy or as an adjunct to stimulant therapy for treatment of attention-deficit/hyperactivity disorder (ADHD) in children ≥6 years old. Clonidine ER tablets have been available for years for treatment of ADHD in children 6-17 years old.

Axatilimab-csfr (Niktimvo – Incyte), a colony stimulating factor-1 receptor-blocking antibody, has been approved by the FDA for intravenous treatment of chronic graft-versus-host disease (cGVHD) after failure of at least 2 lines of systemic therapy in patients weighing ≥40 kg. Corticosteroids are generally the first-line treatment for cGVHD. The oral kinase inhibitors ibrutinib (Imbruvica), ruxolitinib (Jakafi), and belumosudil (Rezurock) are also approved for treatment of cGVHD. Extracorporeal photopheresis has been used off-label.

The FDA has approved Zunveyl (Alpha Cognition), a delayed-release formulation of the prodrug benzgalantamine, for treatment of mild to moderate dementia of Alzheimer's disease (AD). Immediate- and extended-release formulations of the acetylcholinesterase inhibitor galantamine have been available in the US for years for treatment of AD.

Short-acting methylphenidate (MPH) is effective for treatment of attention-deficit/hyperactivity disorder (ADHD), but its 3-5 hour duration of action usually requires mid-day dosing in school, which children may find disruptive or stigmatizing.

Anticoagulants are the drugs of choice for treatment of deep vein thrombosis (DVT) and pulmonary embolism (PE), collectively referred to as venous thromboembolism (VTE).

Disodium pamidronate (Aredia - Ciba-Geigy), an aminohydroxypropilidene bisphosphonate, has been approved by the US Food and Drug Administration for intravenous (IV) treatment of hypercalcemia associated with malignancy, with or without bone metastases. The drug is also being investigated for use in Paget's disease of bone, hyperparathyroidism, and postmenopausal osteoporosis. An oral formulation of pamidronate has been used in Europe, but is not available in the USA.

Rimantadine hydrochloride (Flumadine - Forest), the alpha-methyl derivative of amantadine (Symmetrel, and others), was recently approved by the US Food and Drug Administration for prevention and treatment of influenza A virus infections in adults and for prevention of influenza in children.

Ceftibuten (Cedax - Schering), an oral cephalosporin similar to cefixime (Suprax - Medical Letter, 31:73, 1989), has been approved by the US Food and Drug Administration (FDA) for treatment of acute otitis media, pharyngitis or tonsillitis, and for acute bacterial exacerbations of chronic bronchitis.

Eplerenone (e pler' en one; Inspra - Pharmacia), an aldosterone receptor antagonist similar to spironolactone (Aldactone, and others), has been approved by the FDA, but not yet marketed, for treatment of hypertension. It has also been tried for treatment of heart failure.

Trospium chloride (Sanctura - Indevus/Odyssey) has been approved by the FDA for treatment of overactive bladder with symptoms of urge urinary incontinence, urgency and urinary frequency. It has been available in Europe for many years.