The FDA has approved Dextenza (Ocular Therapeutix), a dexamethasone ophthalmic insert, for treatment of ocular itching associated with allergic conjunctivitis. Dextenza was approved earlier for treatment of ocular inflammation and pain following ophthalmic surgery.

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.

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 sodium-glucose cotransporter 2 (SGLT2) inhibitor empagliflozin (Jardiance – Boehringer Ingelheim/Lilly) is now FDA-approved to reduce the risk of sustained eGFR decline, end-stage kidney disease, cardiovascular death, and hospitalization in adults with chronic kidney disease (CKD) at risk of progression. It is also approved to improve glycemic control in patients ≥10 years old with type 2 diabetes, to reduce the risk of cardiovascular death and hospitalization for heart failure (HF) in adults with HF, and to reduce the risk of cardiovascular death in adults with type...

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).