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).

The FDA has approved a fixed-dose combination of the integrase strand transfer inhibitor (INSTI) elvitegravir, the pharmacokinetic enhancer cobicistat, and the nucleoside/nucleotide reverse transcriptase inhibitors (NRTIs) emtricitabine and tenofovir disoproxil fumarate (Stribild – Gilead) for treatment of HIV-1 infection in antiretroviral treament-naïve adults. Elvitegravir is the second INSTI to be approved by the FDA for use in HIV treatment; raltegravir (Isentress) was the first.

The FDA has approved nusinersen (Spinraza – Biogen) for treatment of spinal muscular atrophy (SMA), a hereditary neurodegenerative disease that occurs in about one in every 10,000 births. It is the first drug to be approved in the US for this indication.

Bicalutamide (Casodex - Zeneca), an oral nonsteroidal antiandrogen similar to flutamide (Eulexin - Medical Letter, 31:72, 1989), is now available in the USA for treat-ment of advanced prostate cancer. The drug is recommended by the manufacturer for use concurrently with an analog of luteinizing hormone-releasing hormone (LHRH) such as leuprolide (Lupron; Lupron Depot) or goserelin (Zoladex -Medical Letter, 32:102, 1990).

Deferasirox (Exjade - Novartis), an oral chelating agent, recently received accelerated approval from the FDA as an orphan drug for oral treatment of chronic iron overload due to blood transfusions (transfusional hemosiderosis) in patients >2 years old. Deferasirox is a tridentate (2 molecules of deferasirox bind to one atom of iron) chelating agent with high affinity for iron. It has much lower affinity for zinc and copper.

Repaglinide (Prandin - Novo Nordisk) is a new oral glucose-lowering agent for treating type 2 diabetes (formerly called non-insulin-dependent diabetes mellitus or NIDDM). It has been approved by the FDA for monotherapy or for use in combination with metformin.

The FDA has approved teduglutide (te due’ gloo tide; Gattex – NPS), a recombinant DNA analog of glucagon-like peptide-2 (GLP-2), for treatment of short bowel syndrome (SBS) in adults who are dependent on parenteral support.

Azacitidine (Vidaza - Pharmion), a pyrimidine nucleoside analog of cytidine, is the first drug approved by the FDA for treatment of myelodysplastic syndrome (MDS). Azacitidine is incorporated into newly synthesized DNA and inhibits DNA methyltransferase. Hypomethylation of DNA can restore the normal expression of genes critical for cell differentiation.

Fludarabine phosphate (Fludara - Berlex), an analog of vidarabine (Vira-A), was recently approved by the U.S. Food and Drug Administration for treatment of patients with refractory chronic lymphocytic leukemia (CLL). The drug has not yet been marketed but is still available, as it has been for two years, through the National Cancer Institute.

Anastrozole (Arimidex - Zeneca), a selective nonsteroidal aromatase inhibitor, has been approved by the US Food and Drug Administration (FDA) for treatment of postmenopausal women with advanced breast cancer that has progressed during treatment with tamoxifen (Nolvadex, and others).