The FDA has approved Xacduro (Innoviva), a combination of the beta-lactam antibacterial sulbactam and the beta-lactamase inhibitor durlobactam, for IV treatment of adults with hospital-acquired or ventilator-associated bacterial pneumonia caused by susceptible isolates of Acinetobacter baumannii-calcoaceticus complex (ABC).

The FDA has approved cantharidin 0.7% solution (Ycanth – Verrica) for topical treatment of molluscum contagiosum in patients ≥2 years old. Ycanth was the first drug to be approved in the US for this indication. A 10.3% gel formulation of berdazimer (Zelsuvmi), a nitric oxide-releasing agent, has also been approved by the FDA for treatment of molluscum contagiosum (in patients ≥1 year old); it will be reviewed in a future issue.

Tovorafenib (Ojemda – Day One), a type II RAF kinase inhibitor, has received accelerated approval from the FDA for treatment of patients ≥6 months old with relapsed or refractory pediatric low-grade glioma harboring a BRAF fusion or rearrangement or a BRAF V600 mutation. Tovorafenib is the first systemic treatment to be approved in the US for pediatric low-grade gliomas with BRAF fusions. Accelerated approval of tovorafenib was based on response rates and duration of response.

The FDA has approved a 0.25% ophthalmic solution of the ectoparasiticide lotilaner (Xdemvy – Tarsus) for treatment of Demodex blepharitis. Lotilaner is the first drug to be approved in the US for this indication.

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 datopotamab deruxtecan (Datroway – Daiichi Sankyo/AstraZeneca), a trophoblast cell-surface antigen-2 (Trop-2)-directed antibody and topoisomerase inhibitor conjugate, for treatment of adults with unresectable or metastatic hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative breast cancer who received prior endocrine-based therapy and chemotherapy for unresectable or metastatic disease.

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.

The FDA has approved cefiderocol (Fetroja – Shionogi), a new IV cephalosporin antibiotic, for treatment of complicated urinary tract infections (UTI) caused by susceptible gram-negative bacteria in adults who have limited or no alternative treatment options.

The FDA has approved lemborexant (Dayvigo – Eisai), an orexin receptor antagonist, for treatment of sleep-onset and/or sleep-maintenance insomnia in adults. It is the second orexin receptor antagonist to be approved for this indication; suvorexant (Belsomra) was the first.