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).

Methylphenidate is now available in a new extended-release formulation (Metadate CD - Celltech) for treatment of attention deficit/hyperactivity disorder (ADHD). The new product, which is a Schedule II controlled substance, is being advertised directly to consumers.

A once-weekly 35-mg oral formulation of the bisphosphonate risedronate (Actonel) has been approved by the FDA for prevention and treatment of postmenopausal osteoporosis. A once-weekly formulation of alendronate (Fosamax) was approved last year (Medical Letter 2001; 43:26). Bisphosphonates bind to the mineral surface of bone and decrease osteoclast activity, inhibiting the resorption phase of the bone turnover cycle. These drugs are not metabolized and remain bound to bone for several weeks.

When the use of aspirin for primary prevention of cardiovascular diseases was last reviewed in The Medical Letter, only one placebo-controlled prospective trial was available: the (male) Physicians' Health Study. Last year, a second large, randomized, placebo-controlled study was reported as part of the Women's Health Study. Recently a sex-specific meta-analysis of 6 trials, including these two, was published.

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The FDA has now approved a recombinant, 9-valent, human papillomavirus (HPV) vaccine (Gardasil 9 – Merck) for use in girls and women 9-26 years old and boys 9-15 years old. The new vaccine is indicated to prevent diseases associated with infection with HPV types 6, 11, 16, 18, 31, 33, 45, 52, and 58, which include genital warts and cervical, vulvar, vaginal, and anal precancerous lesions and cancer. Two recombinant HPV vaccines are already available in the US: Gardasil prevents disease associated with HPV types 6, 11, 16, and 18, and Cervarix prevents disease associated with HPV...

The FDA has approved Bexsero (Novartis/GSK), a vaccine that protects against invasive meningococcal disease caused by Neisseria meningitidis serogroup B. It is the second serogroup B meningococcal vaccine to be approved in the US; Trumenba was the first. Both vaccines are approved for use in persons 10-25 years old. Bexsero is approved in Europe, Canada, and Australia for use in children as young as 2 months old.

The FDA has approved edaravone (Radicava – Mitsubishi Tanabe Pharma) for treatment of amyotrophic lateral sclerosis (ALS). It is the second drug to be approved in the US for this indication; riluzole (Rilutek, and generics) was approved in 1995. Edaravone was approved for treatment of ALS as Radicut in Japan and South Korea in 2015. It has been used in Japan and other Asian countries since 2001 for treatment of acute ischemic stroke.

The FDA has approved a manually injected, single-dose, prefilled epinephrine syringe (Symjepi – Adamis/Sandoz) for emergency treatment of anaphylaxis. The new device is approved in 0.3- and 0.15-mg strengths for treatment of patients weighing ≥30 kg and 15 to 30 kg, respectively; only the 0.3-mg strength is currently available. According to Sandoz, Symjepi will be made available first to institutions and later to the retail market.

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.