A once-weekly transdermal formulation of the acetylcholinesterase inhibitor donepezil (Adlarity – Corium) has been approved by the FDA for treatment of mild, moderate, and severe Alzheimer's disease (AD) dementia. Donepezil is the second acetylcholinesterase inhibitor to become available in a transdermal formulation; transdermal rivastigmine (Exelon Patch), which is applied once daily, has been available for years for the same indication. Donepezil is also available in oral formulations (Aricept, and generics) for treatment of AD dementia.

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.

Betibeglogene autotemcel (Zynteglo — Bluebird Bio), an autologous lentiviral vector cell-based gene therapy, has been approved by the FDA for one-time treatment of transfusion-dependent beta thalassemia in children and adults. Exagamglogene autotemcel (Casgevy), a cell-based gene therapy that uses CRISPR/Cas9 gene-editing technology, was approved earlier this year for the same indication in patients ≥12 years old.

The FDA has approved imetelstat (Rytelo – Geron), a first-in-class telomerase inhibitor, for treatment of low- to intermediate-1 risk myelodysplastic syndromes (MDS) in adults with transfusion-dependent anemia requiring 4 or more red blood cell (RBC) units over 8 weeks who have not responded to, are no longer responding to, or are ineligible for erythropoiesis-stimulating agents (ESAs).

The FDA has approved acoramidis (Attruby – BridgeBio), an oral transthyretin stabilizer, to reduce cardiovascular-related hospitalization and cardiovascular death in adults with wild-type or variant (hereditary) transthyretin amyloid cardiomyopathy (ATTR-CM). Acoramidis is the second transthyretin stabilizer to be approved in the US for this indication; tafamidis (Vyndaqel, Vyndamax) was approved in 2019.

Linvoseltamab (Lynozyfic – Regeneron), a bispecific B-cell maturation antigen (BCMA)-directed CD3 T-cell engager, has received accelerated approval from the FDA for intravenous treatment of relapsed or refractory multiple myeloma in adults who had 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. Linvoseltamab is the third bispecific BCMA-directed CD3 T-cell engager to be approved for this indication; teclistamab...

View the Table: Some Drug Interactions with Drugs for IBD

The FDA has required a new warning in the label of the oral selective neurokinin 3 (NK3) receptor antagonist fezolinetant (Veozah) about the risk of hepatoxicity. The label of fezolinetant, which was approved by the FDA in 2023 for treatment of moderate to severe vasomotor symptoms due to menopause, already contained a warning about hepatic transaminase elevations associated with use of the drug.

The FDA has required a new warning in the label for the scopolamine transdermal patch (Transderm-Scōp, and generics) about a risk of serious or fatal hyperthermia. Scopolamine, an anticholinergic drug, is FDA-approved for prevention of nausea and vomiting associated with motion sickness or recovery after surgery in adults; it is often used off-label to reduce drooling in children and adolescents with neurologic disorders.

The FDA now requires that the labels of all extendedelease stimulants used for treatment of attention-deficit/hyperactivity disorder (ADHD) include a warning about the risks of weight loss and other adverse effects associated with their use in children <6 years old. The labels of all stimulants used for treatment of ADHD already contain a boxed warning about the high risk of abuse and dependence associated with their use.