Octreotide acetate (Sandostatin - Sandoz), a synthetic octapeptide that mimics the actions of somatostatin, was recently approved by the US Food and Drug Administration for symptomatic treatment of patients with metastatic carcinoid or VIPomas (vasoactive intestinal peptide-secreting tumors).

Teniposide (ten i poe' side; VM 26; Vumon - Bristol), an anticancer drug that has been under investigation in the USA for 20 years, has now been approved for use in combination induction treatment of refractory acute lymphoblastic leukemia (ALL) in children. A semisynthetic derivative of podophyllotoxin, teniposide is chemically related to etoposide (VePesid - Medical Letter, 26:48, 1984).

Argatroban, a small-molecule synthetic derivative of L-arginine, is a direct thrombin inhibitor approved by the FDA for use as an anticoagulant in patients with heparin-induced thrombocytopenia (HIT).

Caspofungin acetate(Cancidas) is the first of a new class of antifungals, the echinocandins. It has been approved by the FDA for treatment of invasive aspergillosis in patients who fail to respond to or are unable to tolerate other antifungal drugs such as amphotericin B and itraconazole.

An intraveneous (IV) formulation of ibuprofen (Caldolor - Cumberland) was recently approved by the FDA for use in adults. It can be administered alone for treatment of mild to moderate pain or as an adjunct to opioid analgesics for moderate to severe pain. It is also approved for reduction of fever.

The FDA has approved velaglucerase alfa (Vpriv – Shire), a new formulation of glucocerebrosidase prepared from human fibroblasts, for treatment of the nonneurologic form of Gaucher’s disease (Type 1). Patients with Gaucher’s disease have a congenital deficiency of glucocerebrosidase that leads to accumulation of glucosylceramide, the end-product of sphingolipid catabolism, in the lysozymes of reticuloendothelial cells in the liver, spleen and bone marrow.Velaglucerase is the second form of the enzyme now available in the US; imiglucerase (Cerezyme – Genzyme), which is produced by...

In the past 2 years, the FDA has approved 3 new drugs for prevention or treatment of hereditary angioedema (HAE) in non-pregnant adolescents and adults: the C1 esterase inhibitor Cinryze for prophylaxis and the C1 esterase inhibitor Berinert and the kallikrein inhibitor ecallantide (Kalbitor) for treatment of acute attacks.

Warfarin-related bleeding, especially intracranial hemorrhage, can be catastrophic. Several products are available to reverse warfarin’s anticoagulant effect.

The FDA has approved ferric carboxymaltose (Injectafer – American Regent), a 2-dose intravenous iron replacement product, for treatment of iron deficiency anemia (IDA) in adults who cannot tolerate or have had an unsatisfactory response to oral iron and in those who have non-dialysis-dependent chronic kidney disease (CKD). It is the sixth IV iron product approved in the US.

The antisense oligonucleotide viltolarsen (Viltepso – NS Pharma) has received accelerated approval from the FDA for treatment of Duchenne muscular dystrophy (DMD) in patients who have mutations of the dystrophin gene that are amenable to exon 53 skipping (DMD-53). It is the second drug to be approved for this indication; the antisense oligonucleotide golodirsen (Vyondys 53) was approved in 2019.