Exagamglogene autotemcel (Casgevy – Vertex), a cell-based gene therapy recently approved for treatment of sickle cell disease1, has now been approved by the FDA for treatment of patients ≥12 years old with transfusion-dependent beta thalassemia. Casgevy is the first gene therapy that uses CRISPR/Cas9 gene-editing technology to be approved in the US for any disorder. Betibeglogene autotemcel (Zynteglo), an autologous lentiviral vector cell-based gene therapy, was approved in the US in 2022 for treatment of transfusion-dependent beta thalassemia.

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.

Levocetirizine (Xyzal - UCB/Sanofi-aventis), the active enantiomer of the second-generation H₁-antihistamine cetirizine (Zyrtec), has been approved by the FDA for treatment of seasonal and perennial allergic rhinitis (SAR and PAR) and chronic idiopathic urticaria in adults and children 6 years of age and older. Cetirizine has been approved by the FDA for over-the-counter use and may also become available generically in the US. Levocetirizine has been available in Europe since 2001.

Photopheresis is a new technique recently approved by the US Food and Drug Administration for treatment of cutaneous T-cell lymphoma. It combines oral administration of a photoactivatable cytotoxic drug with leukapheresis and extracorporeal exposure of leukocytes to long-wave ultraviolet light (UVA). This procedure requires use of a 'UVAR Instrument' manufactured by Therakos, a subdivision of Johnson and Johnson.

To avoid the morbidity and occasional mortality of transurethral resection or open prostatectomy, some urologists are now using balloon dilatation of the prostatic urethra to treat benign prostatic hypertrophy.

Gonadorelin (goe nad oh rell'; in) acetate (Lutrepulse - Ortho), a synthetic decapeptide identical in amino acid sequence to human gonadotropin-releasing hormone (GnRH), was recently approved by the US Food and Drug Administration for treatment of primary hypothalamic amenorrhea. Gonadorelin hydrochloride (Factrel) was previously available for diagnostic use (Medical Letter, 25:106, 1983). Analogs of GnRH have been used to treat endometriosis and advanced cancer of the prostate (Medical Letter, 27:71, 1985).

Many readers have asked the Medical Letter to evaluate St. John's wort, an herbal extract now widely sold in health food stores and pharmacies, for its effectiveness and safety in the treatment of depression. St. John's wort is licensed in Germany for treatment of anxiety, depression and insomnia. In the USA, it is considered a dietary supplement and has not been evaluated by the FDA.

A biodegradable polymer 'wafer' (Gliadel - Rh ne-Poulenc Rorer) impregnated with the alkylating agent carmustine (BCNU; Bicnu) has been marketed in the USA for local treatment of recurrent glioblastoma multiforme that requires re-operation. The dime-sized polyanhydride wafers are implanted into the surgical cavity left behind after resection. Release of the alkylating agent directly into the area of the tumor bypasses the blood-brain barrier (H Brem and R Langer, Sci Med, 3:1, 1996).

The US Public Health Service Advisory Committee on Immunization Practices has recently published new recommendations for prevention and control of meningococcal disease in college students.

The FDA has approved marketing of Zegerid powder for oral suspension (Santarus), an immediate-release formulation of the proton-pump inhibitor (PPI) omeprazole (Prilosec, and others). All other oral PPIs are delayed-release, enteric-coated formulations designed to prevent degradation of the drug by gastric acid. Each 20- or 40-mg packet of Zegerid contains 1680 mg of sodium bicarbonate, which protects the drug from gastric acid degradation. A dose of Zegerid contains 460 mg of sodium, which may be excessive for some patients. Zegerid is the first oral PPI to be approved by the FDA for...