Betaine (Cystadane - Orphan Medical), an orphan drug, has recently been marketed for treatment of homocystinuria, a genetic disorder. Patients with homocystinuria frequently have premature atherosclerosis and thromboembolism, and may also have lens dislocations, skeletal abnormalities and mental retardation (SH Mudd et al in CR Scriver et al, eds, The Metabolic and Molecular Bases of Inherited Disease, 7th ed, New York:McGraw Hill, 1995, page 1279).

The standard antithrombotic therapy for treatment of patients with acute coronary syndrome (ACS) is dual antiplatelet therapy with aspirin and clopidogrel (Plavix) or another thienopyridine, plus a parenteral anticoagulant while the patient is hospitalized, followed by antiplatelet therapy alone after discharge. The addition of the oral anticoagulant warfarin (Coumadin, and others) to dual antiplatelet therapy is generally not recommended for this indication because of fluctuations in its anticoagulant effect and the risk of bleeding. A recently published trial found that addition of...

The FDA has approved the oral tyrosine kinase inhibitor cabozantinib (Cabometyx – Exelixis) for treatment of patients with advanced renal cell carcinoma previously treated with antiangiogenic therapy. Cabozantinib was first approved in 2012 as Cometriq for treatment of progressive, metastatic medullary thyroid cancer.Anti-VEGF antibodies, tyrosine kinase inhibitors, and mTOR kinase inhibitors have become the standard of care for treatment of unresectable or metastatic renal cell cancer.1FDA approval was based on the results of a randomized open-label trial (METEOR) comparing cabozantinib to...

The FDA has approved prasugrel (Effient - Lilly/Daiichi Sankyo), an oral antiplatelet drug, for use with aspirin to reduce the rate of thrombotic cardiovascular events in patients with acute coronary syndromes (ACS) being managed with percutaneous coronary intervention (PCI). It will compete with clopidogrel (Plavix) for such use.

Since 2008, because of safety concerns, the FDA has mandated that long-term cardiovascular outcomes trials be conducted for all new drugs for type 2 diabetes. Reductions in the incidence of macrovascular complications in these trials with some sodium-glucose co-transporter 2 (SGLT2) inhibitors and glucagon-like peptide 1 (GLP-1) receptor agonists in patients at risk for cardiovascular disease (see Table 1) have led to new recommendations.

Recent advertisements for indapamide (Lozol - Rorer), an oral antihypertensive/diuretic (Medical Letter, 26:17, 1984), claim that the drug, unlike thiazide diuretics, does not significantly increase serum cholesterol and has a minimal impact on potassium. Indapamide has no thiazide ring but, like chlorthalidone (Hygroton; and others) and hydrochlorothiazide (HydroDiuril; and others), it does have a sulfamoyl benzamide moiety, which is probably responsible for its natriuretic and diuretic effects. Unlike the thiazides, it also has a methylindoline moiety, which apparently decreases...

Adenosine (Adenocard - Fujisawa), an endogenous purine nucleoside, was recently approved by the US Food and Drug Administration for intravenous treatment of paroxysmal supraventricular tachycardia, including Wolff-Parkinson-White (WPW) syndrome. Adenosine is not approved for and has not been effective in treating atrial flutter, atrial fibrillation, or ventricular tachycardias.

Isradipine (DynaCirc - Sandoz), a dihydropyridine calcium-channel blocker chemically related to nicardipine (Cardene) and nifedipine (Procardia, and others), was recently approved by the US Food and Drug Administration for oral treatment of hypertension.

The FDA has required manufacturers of the oral P2Y₁₂ platelet inhibitors clopidogrel (Plavix, and generics), prasugrel (Effient, and generics), and ticagrelor (Brilinta) to warn in the product labels that the absorption of these drugs may be delayed or reduced when taken with an opioid agonist.

The FDA has approved use of Kcentra (CSL Behring), a human-derived 4-factor prothrombin complex concentrate (PCC), for urgent reversal of warfarin anticoagulation in adults with acute major bleeding. It is the only 4-factor PCC available in the US.