Colchicine (Colcrys, and others), which has been available in the US for decades for prophylaxis and treatment of gout flares and other indications in oral formulations that contain 0.6 mg of the drug, has now been approved in 0.5-mg tablets as Lodoco (Agepha) to reduce the risk of myocardial infarction (MI), stroke, coronary revascularization, and cardiovascular death in adults with established atherosclerotic disease or multiple risk factors for cardiovascular disease.

Valoctocogene roxaparvovec-rvox (Roctavian – Biomarin), an adeno-associated virus (AAV) vector-based gene therapy, has been approved by the FDA for one-time treatment of severe hemophilia A in adults without pre-existing antibodies to AAV serotype 5. It is the first gene therapy to be approved in the US for treatment of hemophilia A.

Delandistrogene moxeparvovec-rokl (Elevidys – Sarepta), an adeno-associated virus (AAV) vector-based gene therapy, has received accelerated approval from the FDA for treatment of ambulatory children 4-5 years old with Duchenne muscular dystrophy (DMD) who have a confirmed mutation in the dystrophin gene. It is the first gene therapy to be approved in the US for treatment of DMD. Accelerated approval was based on expression of microdystrophin in skeletal muscle, a surrogate endpoint.

The FDA has approved nirsevimab-alip (Beyfortus – AstraZeneca/Sanofi), a long-acting monoclonal antibody, for the prevention of respiratory syncytial virus (RSV) lower respiratory tract disease in neonates and infants born during or entering their first RSV season, and in children ≤24 months old who are at increased risk for severe RSV disease through their second RSV season. Nirsevimab is the first drug to be approved for protection of all infants during their first RSV season.

The FDA has approved Suflave (Sebela/Braintree), a low-volume polyethylene glycol (PEG)- and sulfate-based product for cleansing of the colon prior to colonoscopy in adults. Other oral colonoscopy preparations available in the US are listed in Table 2. Suflave is marketed as tasting better than other products.

The FDA has approved Opill (Perrigo), a progestin-only oral contraceptive that contains norgestrel, for sale over the counter (OTC). Opill is the first oral contraceptive to be approved in the US for sale without a prescription. Approval of OTC Opill is intended to increase access to effective contraception and reduce the rate of unintended pregnancies.

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...

Opioid use disorder is a chronic, relapsing disease with physical and psychiatric components. It is associated with economic hardship, social isolation, incarceration, increased rates of blood-borne infections such as HIV and viral hepatitis, adverse pregnancy outcomes, and increased mortality. According to the NIH, there were 80,411 deaths involving an opioid in the US in 2021, more than in any previous year. Several guidelines on the management of opioid use disorder are available; all recommend maintenance pharmacotherapy as the standard of care.

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

Akeega (Janssen), a fixed-dose combination of the oral poly(ADP-ribose) polymerase (PARP) inhibitor niraparib (Zejula) and the antiandrogen abiraterone acetate (Zytiga, and others), has been approved by the FDA for use in combination with prednisone for treatment of adults with deleterious or suspected deleterious BRCA-mutated (BRCAm) metastatic castration-resistant prostate cancer (mCRPC). Niraparib has been available since 2017 for treatment of epithelial ovarian, fallopian tube, or primary peritoneal cancer. Abiraterone acetate has been available since 2011 for treatment of CRPC...