Tegaserod maleate (Zelnorm – Novartis), a partial serotonin 5-HT4 receptor agonist that increases gastrointestinal motility, was approved by the FDA in 2002 for short-term treatment of constipation-predominant irritable bowel syndrome in women,1 and in 2004 for treatment of chronic constipation in adults ≤65 years old. Its efficacy has not been impressive statistically, but according to Medical Letter consultants some patients with slow-transit constipation have benefited from taking the drug. Diarrhea has been its main adverse effect.2The FDA now has requested that the manufacturer stop...

Transcatheter aortic-valve replacement (TAVR), a procedure in which a prosthetic valve is inserted into the aortic annulus by a catheter, has been approved by the FDA as an alternative to surgical aortic-valve replacement for patients with severe symptomatic aortic stenosis who are considered inoperable.

The FDA has approved an oral liquid formulation of glycerol phenylbutyrate (Ravicti – Hyperion) for chronic management of patients ≥2 years old with urea cycle disorders that cannot be adequately managed by a protein- restricted diet. Sodium phenylbutyrate (Buphenyl, and generics), another oral drug approved by the FDA for this indication, has a bitter taste. The new product, which contains no sodium, has little or no taste. Either drug must be used in addition to a protein-restricted diet and, if needed, dietary supplementation with amino acids and other nutrients.

Risdiplam (Evrysdi – Genentech), a survival of motor neuron 2 (SMN2) splicing modifier, has been approved by the FDA for oral treatment of spinal muscular atrophy (SMA) in patients ≥2 months old. It is the first oral drug to be approved in the US for treatment of SMA; nusinersen (Spinraza), an intrathecally administered SMN2-directed antisense oligonucleotide, and onasemnogene abeparvovec (Zolgensma), an IV adeno-associated virus vector-based gene therapy, were approved earlier.

Difelikefalin (Korsuva – Vifor), an IV kappa opioid receptor (KOR) agonist, has been approved by the FDA for treatment of moderate to severe pruritus associated with chronic kidney disease (CKD) in adults on hemodialysis. It is the first drug to be approved for this indication and the first KOR agonist to become available in the US. Difelikefalin has not been studied in patients on peritoneal dialysis.

The Bruton's tyrosine kinase (BTK) inhibitor pirtobrutinib (Jaypirca – Lilly) has received accelerated approval from the FDA for treatment of relapsed or refractory mantle cell lymphoma (MCL) in adults who received ≥2 prior lines of systemic therapy, including a BTK inhibitor. Accelerated approval was based on the response rate. The BTK inhibitors ibrutinib (Imbruvica), acalabrutinib (Calquence), and zanubrutinib (Brukinsa) are also approved for treatment of MCL.

Elranatamab-bcmm (Elrexfio – Pfizer), a bispecific B-cell maturation antigen (BCMA)-directed CD3 T-cell engager, has been granted accelerated approval by the FDA for treatment of relapsed or refractory multiple myeloma in adults who received ≥4 prior lines of therapy, including a proteasome inhibitor, an immunomodulatory drug, and an anti-CD38 monoclonal antibody. Accelerated approval was based on the response rate and durability of response. Elranatamab is the second bispecific BCMA-directed CD3 T-cell engager to be approved for this indication; teclistamab (Tecvayli) was the...

The FDA has approved Azmiro (Azurity), the first injectable formulation of testosterone cypionate to become available in single-dose vials and prefilled syringes for treatment of males with conditions associated with a deficiency or absence of endogenous testosterone. Injectable testosterone cypionate has been available in multidose vials (Depo-Testosterone, and generics) for many years. Testosterone enanthate (Xyosted) is available in prefilled autoinjectors for use in adult men. No testosterone products are approved for treatment of low testosterone levels due solely to aging. All...

Gamma hydroxy butyrate (GHB) sold in health food stores has recently caused outbreaks of gastrointestinal illness, central-nervous-system (CNS) depression, and seizures.

The FDA has approved the marketing of RotaShield, a live oral vaccine for prevention of infantile rotavirus infection. Rotaviruses are segmented double-stranded RNA viruses that are the most common cause of severe diarrhea and dehydration in infants and young children.