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...

View Comparison Table: Some Drugs for Maintenance Treatment of Opioid Use Disorder

Two cell-based gene therapies – exagamglogene autotemcel (Casgevy – Vertex) and lovotibeglogene autotemcel (Lyfgenia – Bluebird Bio) – have been approved by the FDA for treatment of sickle cell disease in patients ≥12 years old with recurrent vaso-occlusive crises. They are the first gene therapies to be approved in the US for use in sickle cell disease; Casgevy is the first treatment to be approved in the US that uses CRISPR/Cas9 gene-editing technology.

Tapinarof 1% cream (Vtama – Dermavant), an aryl hydrocarbon receptor (AhR) agonist, has been approved by the FDA for topical treatment of atopic dermatitis in patients ≥2 years old. Tapinarof is the first AhR agonist to be approved in the US for this indication. It was approved in 2022 for treatment of plaque psoriasis in adults.

Nicardipine (Cardene - Syntex), a dihydropyridine calcium-entry blocker structurally related to nifedipine (Procardia; Adalat), was recently approved by the US Food and Drug Administration for oral treatment of angina and hypertension. Advertisements for the drug claim that nicardipine is more vasoselective; than other calcium-channel blockers and does not depress myocardial contractility.

Taking aspirin to prevent recurrence or worsening of cardiovascular disease has become a common practice in recent years (Medical Letter, 28:31, 1986). Now, the US Food and Drug Administration is considering whether aspirin can also be advertised for prevention of a first myocardial infarction in previously healthy people.

Omeprazole (Losec - Merck), a new drug that suppresses gastric acid secretion, has been approved by the US Food and Drug Administration for short-term (four to eight weeks) treatment of severe or refractory gastroesophageal reflux and for long-term treatment of pathological hypersecretory conditions such as Zollinger-Ellison syndrome, multiple endocrine adenomas, and systemic mastocytosis. Although widely used abroad for treatment of peptic ulcers, it has not been approved for that indication in the USA.

Sumatriptan (soo ma trip' tan; Imitrex - Glaxo), a serotonin (5-HT) agonist, is now available in Canada and may soon be available in the USA for oral or parenteral treatment of migraine headache. The parenteral formulation is designed for patients to inject themselves subcutaneously. Sumatriptan is not recommended for prophylactic use.

Cisapride (Propulsid - Janssen), a piperidinyl benzamide prokinetic drug, is now available in the USA for symptomatic relief of nocturnal heartburn due to gastroesophageal reflux disease. In Canada, cisapride is also marketed for treatment of gastroparesis.

Recent reports have suggested that some obese patients may benefit from long-term pharmacologic treatment (RL Atkinson and VS Hubbard, Am J Clin Nutr, 60:153, August 1994; DJ Goldstein and JH Potvin, Am J Clin Nutr, 60:647, November 1994). Fluoxetine (Prozac), widely used for treatment of depression and near approval by the US Food and Drug Administration (FDA) for treatment of bulimia, is also being tried for treatment of obesity.