The FDA has approved an auto-injector formulation of testosterone enanthate (Xyosted – Antares Pharma) for once-weekly subcutaneous self-administration in adult males with conditions associated with a deficiency or absence of endogenous testosterone. It is the first subcutaneous formulation of testosterone to be approved by the FDA. Xyosted is contraindicated for treatment of low testosterone levels associated with aging.

Odevixibat (Bylvay – Albireo), an oral ileal bile acid transporter (IBAT) inhibitor, has been approved by the FDA for treatment of pruritus in patients ≥3 months old with progressive familial intrahepatic cholestasis (PFIC). It is the first drug to be approved in the US for this indication.

Amivantamab-vmjw (Rybrevant – Janssen), an EGFR-MET bispecific antibody, has received accelerated approval from the FDA for IV treatment of locally advanced or metastatic non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 20 insertion mutations in adults whose disease has progressed on or after platinum-based chemotherapy. It is the first bispecific antibody to become available in the US for this indication. Accelerated approval of the drug was based on the overall response rate and duration of response.

Berdazimer 10.3% gel (Zelsuvmi – Ligand), a first-in-class nitric oxide-releasing agent, has been approved by the FDA for topical treatment of molluscum contagiosum in patients ≥1 year old. It is the second drug to become available in the US for treatment of molluscum contagiosum and the first that can be applied by the patient or caregiver at home; cantharidin 0.7% solution (Ycanth), which is applied by a healthcare professional, was approved earlier for use in patients ≥2 years old.

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 oral Janus kinase (JAK) inhibitor upadacitinib (Rinvoq – Abbvie) has been approved by the FDA for treatment of giant cell arteritis. Upadacitinib was approved earlier for treatment of rheumatoid arthritis, psoriatic arthritis, ankylosing spondylitis, nonradiographic axial spondyloarthritis, atopic dermatitis, ulcerative colitis, and Crohn's disease.

The recent terrorist attacks on the US have led to many questions about the clinical effects, prevention and treatment of injury caused by chemical warfare agents.

The FDA has expanded the Emergency Use Authorizations (EUAs) for the mRNA-based COVID-19 vaccines manufactured by Pfizer/BioNTech (Comirnaty) and Moderna (Spikevax) and the adenovirus-based vaccine manufactured by Johnson & Johnson/Janssen to include administration of a booster dose in select populations after primary immunization with either the same COVID-19 vaccine or a different one.

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.