Efalizumab (Raptiva - Genentech/XOMA), a humanized monoclonal antibody that inhibits T-cell activation, has been approved by the FDA for treatment of adults with moderate to severe chronic plaque psoriasis.

Advertisements are appearing in medical journals for continuing medical education courses on catheter ablation of atrial fibrillation. Is a cure at hand for this common disorder?

A reader has asked us to review the choice of drugs for heparin-induced thrombocytopenia (HIT), a prothrombotic state with high morbidity and mortality. Three recent publications have made somewhat different recommendations on its treatment.

The FDA has approved pembrolizumab (Keytruda – Merck), a human programmed death receptor-1 (PD-1) blocking antibody, for treatment of unresectable or metastatic melanoma that has progressed following treatment with ipilimumab (Yervoy) and, if the patient is BRAF V600 mutation positive, a BRAF inhibitor. It is the fi rst PD-1 inhibitor to be marketed in the US. Nivolumab, another PD-1 inhibitor, is available in Japan. Pembrolizumab was previously known as lambrolizumab.

The FDA has approved eliglustat (Cerdelga – Genzyme), an oral glucosylceramide synthase inhibitor, for treatment of adults with type 1 Gaucher disease. Eliglustat is metabolized primarily by CYP2D6. Because patients who are CYP2D6 ultra-rapid metabolizers may not achieve therapeutic concentrations and a specific dosage cannot be recommended for indeterminate metabolizers, the FDA has approved the drug only for patients who are extensive, intermediate, or poor metabolizers of CYP2D6.

Tofersen (Qalsody – Biogen), an intrathecally administered antisense oligonucleotide, has received accelerated approval from the FDA for treatment of amyotrophic lateral sclerosis (ALS) in adults who have a mutation in the superoxide dismutase 1 (SOD1) gene. About 2% of patients with ALS have mutations in the SOD1 gene. Tofersen is the first drug to be approved in the US that targets a genetic cause of ALS. Accelerated approval of the drug was based on the surrogate endpoint of a reduction in plasma neurofilament light chain, which is considered likely to predict clinical...

Daptomycin (Cubicin - Cubist), a cyclic lipopeptide (a new class of antibiotics), has been approved by the FDA for intravenous (IV) treatment of complicated skin and skin structure infections. It is being promoted as a once-daily bactericidal alternative to vancomycin (Vancocin, and others) for methicillin-resistant Staphylococcus aureusinfections.

The FDA has approved a new formulation of dexrazoxane (Totect) for treatment of extravasation from intravenous (IV) anthracyclines such as doxorubicin (Adriamycin, and others). Dexrazoxane has been available since 1995 as Zinecard for protection against the cardiac toxicity of anthracyclines (Med Lett Drugs Ther 1995; 37:110). It is also available generically. The drug’s precise mechanism of action is not known, but anthracyclines are vesicants that bind to DNA and act as oxidizing agents in the presence of iron. Dexrazoxane is a topoisomerase inhibitor, possibly interfering with...

The FDA has approved the intravenously administered synthetic vasopressin analog terlipressin (Terlivaz – Mallinckrodt) to improve kidney function in adults with hepatorenal syndrome (HRS) and a rapid decline in kidney function. Terlipressin is the first drug to be approved for this indication in the US; it has been available in Europe and elsewhere for year

The FDA has approved Vowst (Seres Therapeutics/Nestle HealthScience), an oral capsule containing live fecal microbiota spores, for prevention of additional recurrences of Clostridioides difficile infection (CDI) in adults. Vowst is the first orally administered microbiota-based treatment to be approved for this indication. A rectally-administered live fecal microbiota-based suspension (Rebyota) was approved in 2022 for the same indication. Neither product is approved for acute treatment of CDI.