The results of the recently published FOURIER trial have shown a reduction in cardiovascular events with addition of the PCSK9 inhibitor evolocumab (Repatha) to statin therapy in patients with atherosclerotic cardiovascular disease (ASCVD).

Lisdexamfetamine dimesylate (Vyvanse - Shire), a prodrug in which d-amphetamine is covalently bonded to L-lysine, has been approved by the FDA for treatment of attention-deficit/hyperactivity disorder (ADHD) in children 6-12 years old. It was designed to have less potential than amphetamine itself for abuse, diversion or overdose toxicity. Like methylphenidate and amphetamines, lisdexamfetamine is a Schedule II controlled substance.

The FDA has approved a transmucosal tablet formulation of fentanyl citrate (Fentora - Cephalon) for management of breakthrough pain in opioid-tolerant patients with cancer. An oral transmucosal fentanyl lozenge on a stick (Actiq, and others) is also available for this indication, and is widely used off-label for chronic, non-cancer pain as well.

Mitapivat (Aqvesme – Agios), an oral pyruvate kinase activator, has been approved by the FDA for treatment of anemia in adults with alpha or beta thalassemia. It is the first product to be approved in the US for treatment of alpha thalassemia and the first oral option for beta thalassemia. Mitapivat was approved in 2022 as Pyrukind for treatment of hemolytic anemia in adults with pyruvate kinase deficiency.

The sodium-glucose cotransporter 2 (SGLT2) inhibitor dapagliflozin (Farxiga – AstraZeneca) was approved by the FDA in 2020 to reduce the risk of cardiovascular death and hospitalization for heart failure (HF) in adults with heart failure with reduced ejection fraction (HFrEF). The indication has now been expanded to include a reduction in the risk of urgent HF visits and use in adults with any left ventricular ejection fraction (LVEF).

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.

Testing for drug use has become increasingly common, not only in health care, but also in drug rehabilitation, in the military, at the workplace, after accidents and in the criminal justice system. Performance-enhancing drugs such as anabolic steroids, growth hormone (Genotropin, and others) and erythropoietin (Procrit, Epogen) are not discussed here.

The FDA has approved mavacamten (Camzyos – MyoKardia/BMS), a modulator of cardiac myosin, to improve functional capacity and symptoms in adults with New York Heart Association (NYHA) class II or III obstructive hypertrophic cardiomyopathy (HCM) who have a baseline left ventricular ejection fraction (LVEF) ≥55%. Mavacamten is the first drug in its class to become available in the US.

Pancreatic enzyme replacement products have been used for years to improve digestion in patients with exocrine pancreatic insufficiency (EPI). These products were initially marketed before formal FDA approval was required; in 1991, however, the FDA told all manufacturers of pancreatic enzyme replacement products that they would have to submit a new drug application by April 2010 in order to keep their products on the market.EPI is a chronic disorder characterized by a deficiency of exocrine pancreatic enzymes, which results in malabsorption, steatorrhea, and weight loss. Irreversible EPI is...

The FDA has approved Cardamyst (Milestone), a nasal spray formulation of the nondihydropyridine calcium channel blocker etripamil, for conversion of acute symptomatic episodes of paroxysmal supraventricular tachycardia (PSVT) to sinus rhythm in adults. Cardamyst is the first product containing etripamil to be approved in the US. It is being marketed as the only FDA-approved, self-administered, rapid-acting treatment option for acute episodes of PSVT.