The FDA has approved use of dinutuximab (Unituxin [yoo ni tux' in] – United Therapeutics) in combination with interleukin-2 (IL-2), granulocyte-macrophage colony-stimulating factor (GM-CSF), and isotretinoin for treatment of children with high-risk neuroblastoma who previously responded to first-line therapies. Dinutuximab is a monoclonal antibody that binds to GD2, a glycolipid that is overexpressed on the surface of neuroblastoma cells.1Dinutuximab received a priority review and orphan drug designation. Approval was based on the results of an open-label trial in 226 patients with high-risk...

The FDA has approved oral cholic acid (Cholbam – Retrophin) for treatment of children and adults with bile acid synthesis disorders caused by single enzyme defects and for adjunctive treatment of peroxisomal disorders such as Zellweger spectrum disorders in patients who have liver disease, steatorrhea, or complications from fat-soluble vitamin malabsorption. Patients with these rare inborn errors of bile acid metabolism cannot synthesize primary bile acids such as cholic acid, resulting in reduced bile flow, decreased absorption of fat and fat-soluble vitamins, and development of liver...

The FDA has required that the labels of all carbidopa/levodopa-containing products (Crexont, Dhivy, Duopa, Rytary, Sinemet, Sinemet CR, Stalevo, Vyalev) include a warning about the risk of vitamin B6 (pyridoxine) deficiency and vitamin B6 deficiency-related seizures associated with their use. The combination of carbidopa/levodopa is the most effective treatment for the motor symptoms of Parkinson's disease and recent evidence favors its early use.

Intravenous formulations of immune globulin (IVIG) have been available for more than ten years for treatment of immune deficiency (Medical Letter, 24:81, 1982). Seven preparations are now licensed in the USA, with additional indications for their use.

Four new drugs have become available in the USA for HIV infection or its complications. Nevirapine (Viramune - Roxane), intravenous cidofovir (Vistide - Gilead), and amphotericin B oral suspension (Fungizone Oral Suspension - Bristol-Myers Squibb) have been approved by the US Food and Drug Administration; nelfinavir (Viracept - Agouron) is currently available only through an "Expanded Access Program."

Oxcarbazepine, which is chemically similar to carbamazepine, and levetiracetam, a pyrrolidine acetamide chemically unrelated to other antiepileptic drugs, are the sixth and seventh drugs approved by the US Food and Drug Administration in the last five years for oral use in partial seizures.

Pantoprazole, the fourth benzimidazole proton pump inhibitor to become available in the United States, has been marketed for short-term oral treatment of erosive gastroesophageal reflux disease (GERD).

A recent supplement to the Journal of Clinical Psychiatry, titled "The Expert Consensus Guideline Series: Optimizing Pharmacologic Treatment of Psychotic Disorders," concluded that most experts endorsed use of second-generation (atypical) antipsychotics rather than first-generation drugs, with risperidone (Risperdal - Janssen) the top choice for first episodes, multiple episodes or switches from another drug (JM Kane et al, J Clin Psychiatry 2003; 64 suppl 12:5). The supplement was sponsored by Janssen.

Rifaximin (Xifaxan - Salix), a non-absorbed oral antibiotic derived from rifampin (Rifadin, and others), has been approved by the FDA for treatment of travelers' diarrhea caused by noninvasive strains of Escherichia coli in patients 12 years of age or older. It has been available in Europe since 1987.

A fixed-dose combination of oxycodone 5 mg and ibuprofen 400 mg (Combunox - Forest Laboratories) is now available and is being heavily advertised for short-term treatment of moderate to severe acute pain.