Berotralstat (Orladeyo – Biocryst), an oral plasma kallikrein inhibitor, has been approved by the FDA for prevention of hereditary angioedema (HAE) attacks in adults and children ≥12 years old. The subcutaneously-injected plasma kallikrein inhibitor lanadelumab-flyo (Takhzyro) and the human plasma-derived C1 esterase inhibitors (C1INHs) Cinryze, which is given IV, and Haegarda, which is given SC, have been available for prophylaxis of HAE for years.

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.

The FDA has approved cannabidiol oral solution (Epidiolex – Greenwich Biosciences) for treatment of seizures associated with Dravet syndrome or Lennox-Gastaut syndrome in patients ≥2 years old. Cannabidiol (CBD) is a cannabinoid constituent of the marijuana plant (Cannabis sativa). It is the first natural marijuana product to be approved by the FDA for any indication and the first drug to be approved in the US for treatment of Dravet syndrome. Stiripentol (Diacomit), which is not a marijuana product, was also recently approved by the FDA for treatment of Dravet syndrome in...

Antiretroviral therapy is recommended for all HIV-infected patients, both to reduce the risk of disease progression and to prevent transmission of the virus to others. Various guidelines for treatment of HIV infection are available.

The FDA has approved over-the-counter (OTC) sale of the weight-loss drug orlistat (Xenical – Roche)1 as alli (GlaxoSmithKline), to be used in combination with a reduced-calorie, low-fat diet. Orlistat binds to gastric and pancreatic lipases, preventing absorption of about 30% of dietary fat with the 120-mg prescription dose, and 25% with the 60-mg OTC dose. A 16-week controlled trial of the OTC dose (60 mg 3 times daily with meals) in overweight, not obese, patients (average BMI 26.8) on a reduced-calorie, low-fat diet found that patients taking the drug lost 1.15 kg more than those taking...

Itraconazole (Sporanox - Janssen), an oral antifungal triazole that has been available in the USA since 1992 for treatment of deep fungal infections (Medical Letter, 35:7, 1993), is now being marketed for treatment of dermatophyte infections of the toenails, with or without fingernail involvement.

The US Food and Drug Administration (FDA) has approved two new retinoids for treatment of psoriasis. Tazarotene gel 0.05% and 0.1% (Tazorac - Allergan) is being marketed for topical use in patients with stable, mild to moderate plaque psoriasis. Acitretin (Soriatane - Roche) is being promoted for systemic use in severe psoriasis.

The FDA has approved rilpivirine (Edurant – Janssen), a non-nucleoside reverse transcriptase inhibitor (NNRTI), for use with other antiretroviral agents for treatment of HIV-1 infection in treatment-naive adults. Rilpivirine is also available in a fixed-dose combination with emtricitabine and tenofovir (Complera – Gilead).

Rasagiline (Azilect - Teva), a monoamine oxidase-type B (MAO-B) inhibitor, was recently approved by the FDA for once-daily oral treatment of Parkinson's disease (PD). It can be taken alone for treatment of early disease or with levodopa/carbidopa (Sinemet, and others) for advanced disease. Selegiline (Eldepryl, and others), the first MAO-B inhibitor marketed in the US, has been available since 1988; a new lower-dose disintegrating tablet (Zelapar) was recently approved.

The FDA has approved a new tablet formulation of bromocriptine mesylate (Cycloset – VeroScience) for treatment of type 2 diabetes in adults. Bromocriptine (Parlodel, and others) is an ergot-derived dopamine agonist that has been used for more than 20 years to treat hyperprolactinemia, acromegaly, Parkinson’s disease and restless leg syndrome.