Nadofaragene firadenovec-vncg (Adstiladrin – Ferring), an adenoviral vector-based gene therapy, has been approved by the FDA for treatment of adults with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ with or without papillary tumors. It is the first adenoviral vector-based gene therapy to be approved in the US for this indication. The immune checkpoint inhibitor pembrolizumab (Keytruda) was approved for the same indication in 2021.

Nogapendekin alfa inbakicept-pmln (Anktiva – ImmunityBio), a first-in-class interleukin-15 (IL15) receptor agonist, has been approved by the FDA for use with Bacillus Calmette-Guérin (BCG) for treatment of patients with BCG-unresponsive nonmuscle invasive bladder cancer (NMIBC) with carcinoma in situ with or without papillary tumors. Such patients generally undergo bladder tumor resection, followed by intravesical BCG treatment, but treatment failure and cancer recurrence are common. The adenoviral vector-based intravesical gene therapy nadofaragene firadenovec-vncg (Adstiladrin)...

Anticonvulsants are now widely used for treatment of psychiatric illnesses, particularly bipolar disorder. Lithium is the standard drug for treatment of bipolar disorder, but it can cause severe toxicity, serum concentrations must be monitored, and it is not effective in some patients.

Rifaximin (Xifaxan - Salix), a non-absorbed oral antibiotic derived from rifampin (Rifadin, and others), has been approved by the FDA for treatment of travelers' diarrhea caused by noninvasive strains of Escherichia coli in patients 12 years of age or older. It has been available in Europe since 1987.

Gamma hydroxybutyrate (GHB), a central-nervous-system depressant notorious for its use in "date rape", was approved by the FDA in 2002 as sodium oxybate (Xyrem - Jazz Pharmaceuticals) for oral treatment of cataplexy in patients with narcolepsy. Now it has also been approved for treatment of excessive daytime sleepiness (EDS) in these patients. Xyrem is a Schedule III controlled substance.

Conivaptan hydrochloride (Vaprisol - Astellas), a vasopressin antagonist, has been approved by the FDA for short-term intravenous (IV) treatment of euvolemic hyponatremia in hospitalized patients. Euvolemic hyponatremia is most often caused by the syndrome of inappropriate ADH secretion (SIADH), or by hypothyroidism or adrenal insufficiency. The drug has not been approved for treatment of hypervolemic hyponatremia, which is associated with congestive heart failure (CHF), cirrhosis and renal disease. It should not be used in hypovolemic hyponatremia, such as occurs when fluid losses are...

The FDA has approved the sGC stimulator riociguat (rye" oh sig' ue at; Adempas – Bayer) for oral treatment of pulmonary arterial hypertension (PAH) and chronic thromboembolic pulmonary hypertension (CTEPH) following surgery or when surgery is not an option. It is the first drug to be approved for treatment of CTEPH.

The FDA has approved peramivir (Rapivab – BioCryst), an IV neuraminidase inhibitor administered as a single dose, for treatment of acute uncomplicated influenza in patients ≥18 years old who have had symptoms for no more than 2 days. Peramivir was available temporarily in the US during the 2009-2010 influenza season under an emergency use authorization for treatment of hospitalized patients. It has been available in some Asian countries since 2010. Peramivir is the third neuraminidase inhibitor to be approved in the US. Oseltamivir (Tamiflu), which is taken orally, and zanamivir...

The FDA has approved a second extended-release (ER) formulation of the oral opioid agonist hydrocodone (Hysingla ER – Purdue) for management of pain severe enough to require continuous long-term therapy and for which alternative treatment options are inadequate. Hysingla ER tablets have abuse-deterrent properties to discourage their misuse.

The FDA has approved naloxegol (Movantik – AstraZeneca), a pegylated derivative of the opioid antagonist naloxone, for oral treatment of opioid-induced constipation in adults with chronic noncancer pain. It is the only oral opioid antagonist approved for this indication in the US.