The FDA has approved pitolisant (Wakix – Harmony), a histamine-3 (H₃)-receptor antagonist/inverse agonist, for treatment of excessive daytime sleepiness (EDS) or cataplexy in adults with narcolepsy. It is the first H₃-receptor antagonist/inverse agonist to become available in the US and the first non-controlled substance to receive FDA approval for these indications. Pitolisant has been available in Europe since 2016.

Atrial fibrillation (AF) is the most common arrhythmia in the world. Risk factor modification, anticoagulation, rhythm control, and rate control are the four pillars of its management. American College of Cardiology/American Heart Association (ACC/AHA) guidelines on management of AF were updated recently.

Overweight is generally defined as a body mass index (BMI) between 25 and 29.9 kg/m2 in adults. A BMI ≥30 is considered obese (criteria differ slightly in Asian patients). In patients with an elevated BMI, a 5-10% reduction in weight has been associated with a reduction in the risk of developing type 2 diabetes, hypertension, and dyslipidemia. Diet, exercise, and behavior modification are the traditional methods for losing weight, but they are often inadequate; the American College of Cardiology no longer recommends that all patients try lifestyle modification alone before adding...

The FDA has approved a 0.016% gel formulation of the nitrogen mustard mechlorethamine (Valchlor – Actelion) for second-line topical treatment of patients with stage IA or IB mycosis fungoides, the most common type of cutaneous T-cell lymphoma. Topical nitrogen mustard has been used off-label for decades for this indication, usually as a pharmacy-compounded ointment, but no clinical trials evaluating its efficacy and safety have been done. Mechlorethamine is also available in an injectable formulation (Mustargen – Recordati) for the same and other indications.FDA approval of Valchlor was...

In 2007, an FDA advisory committee asked that placebo-controlled, dose-ranging trials be conducted to establish the efficacy of the oral decongestant phenylephrine (Sudafed PE, and others), which is sold over the counter (OTC) as a single agent and in combination with other drugs for treatment of cold and allergy symptoms. Phenylephrine replaced pseudoephedrine (Sudafed, and others) in many OTC formulations when access to pseudoephedrine-containing products was restricted in an effort to reduce their use in the synthesis of methamphetamine.CLINICAL STUDIES — In a randomized, open-label,...

The FDA has approved Lonsurf (Taiho Oncology), a combination of the thymidine-based nucleoside analog trifluridine and the thymidine phosphorylase inhibitor tipiracil, for oral treatment of metastatic colorectal cancer. Trifluridine is incorporated into DNA, interfering with DNA synthesis and inhibiting cell proliferation. Tipiracil inhibits the metabolism of trifluridine. The combination is only approved for use in patients who were previously treated with a fluoropyrimidine (fluorouracil or capecitabine), oxaliplatin, irinotecan, an anti-VEGF biological such as bevacizumab, and, if the tumor...

The FDA has approved an oral suspension formulation of nitisinone (Orfadin – Sobi) for treatment of hereditary tyrosinemia type 1 (HT-1). Orfadin has been available in a capsule formulation in Canada since 1994 and in the US since 2002.HT-1 is an autosomal recessive genetic disorder (estimated birth prevalence: 1:100,000) caused by mutations in the gene responsible for formation of fumarylacetoacetate hydrolase (FAH), which catalyzes the last step in tyrosine metabolism. FAH deficiency results in accumulation of upstream toxic metabolites, causing liver and kidney failure and developmental...

The FDA has approved adjunctive treatment with Dartisla ODT (Edenbridge), a new orally disintegrating tablet (ODT) formulation of the anticholinergic drug glycopyrrolate, to reduce symptoms of a peptic ulcer. Glycopyrrolate oral tablets (Robinul, Robinul Forte, and generics) were approved for the same indication in 1961.

Six vaccines are recommended for routine use in adults living in the USA (Guide for Adult Immunization, 2nd ed, Philadelphia:American College of Physicians, 1990). Immunization recommendations for travel outside the USA were published in The Medical Letter, volume 32, page 33, April 6, 1990.

Two new drugs alendronate (Fosamax - Merck) and salmon calcitonin nasalspray (Miacalcin - Sandoz) are now available in the USA for treatment of postmenopausalosteoporosis. A third drug for treatment of osteoporosis, a slow-release fluoridepreparation (Slow Fluoride -Mission Pharmacal), has been recommended for approvalby an advisory committee of the US Food and Drug Administration (FDA). Various formulationsof fluoride have been available in Europe for this indication for many years.