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.

The oral kinase inhibitor osimertinib (Tagrisso – AstraZeneca), which has been available for years for treatment of non-small cell lung cancer (NSCLC) in adults with epidermal growth factor receptor (EGFR) exon 19 deletions or exon 21 L858R mutations, has now been approved for treatment of unresectable stage III EGFR-mutated NSCLC. About 20-30% of patients with NSCLC have locally advanced stage III NSCLC, and 60-90% of these patients have unresectable disease. Osimertinib is the first targeted therapy to be approved for the new indication.

Zolbetuximab-clzb (Vyloy – Astellas), a claudin 18.2 (CLDN18.2)-directed cytolytic antibody, has been approved by the FDA for use in combination with fluoropyrimidine- and platinum-containing chemotherapy for first-line treatment of locally advanced unresectable or metastatic human epidermal growth factor receptor 2 (HER2)-negative gastric or gastroesophageal junction (GEJ) adenocarcinoma in patients who have CLDN18.2-positive tumors. It is the first monoclonal antibody to be approved in the US that selectively targets CLDN18.2.

Plozasiran (Redemplo – Arrowhead), an apolipoprotein C-III (apoC-III)-directed small interfering ribonucleic acid (siRNA), has been approved by the FDA to reduce triglyceride levels in adults with familial chylomicronemia syndrome (FCS). It is the second drug to be approved in the US for this indication; the apoC-III-directed antisense oligonucleotide olezarsen (Tryngolza) was approved in 2024.

The oral kinase inhibitors selpercatinib (Retevmo – Lilly) and pralsetinib (Gavreto – Blueprint Medicines/Genentech) have been approved by the FDA for treatment of advanced or metastatic RET-mutant or RET fusion-positive cancers. They are the first drugs to be approved specifically for certain RET-driven cancers. Both drugs were granted accelerated approval based on overall response rates and the duration of response.

Resmetirom (Rezdiffra – Madrigal), a thyroid hormone receptor-beta agonist, has received accelerated approval from the FDA for treatment of noncirrhotic nonalcoholic steatohepatitis (NASH) with moderate to advanced fibrosis in adults. NASH has recently been renamed metabolic dysfunction-associated steatohepatitis (MASH). Resmetirom is the first drug to be approved in the US for this indication.

When used for the appropriate seizure type, antiseizure medications (ASMs) are roughly equivalent in efficacy. In addition to the seizure type, the choice of drug is usually based on factors such as ease of use, spectrum of activity, adverse effects, interactions with other drugs, presence of comorbid conditions, suitability for elderly persons and those with childbearing potential, and cost. Treatment should begin with a single drug, increasing the dosage gradually until seizures are controlled or adverse effects become unacceptable. If seizures persist, expert clinicians generally...

Anticoagulants are the drugs of choice for treatment and prevention of deep venous thrombosis (DVT) and pulmonary embolism (PE), collectively referred to as venous thromboembolism (VTE). US guidelines for treatment of VTE were updated in 2020 and 2021.

Cholesterol management guidelines from the American College of Cardiology/American Heart Association Task Force were last published in 2019.

The sodium-glucose cotransporter 2 (SGLT2) inhibitor empagliflozin (Jardiance – Boehringer Ingelheim) was approved by the FDA in 2021 to reduce the risk of hospitalization for heart failure (HF) and cardiovascular death in patients with heart failure with reduced ejection fraction (HFrEF; LVEF ≤40%), regardless of whether or not they have type 2 diabetes. The indication has now been expanded to include patients with HF with any ejection fraction. Empagliflozin is the first SGLT2 inhibitor to be approved in the US for this indication.