The FDA has approved Trikafta (Vertex), a fixed-dose combination of the cystic fibrosis transmembrane conductance regulator (CFTR) modulators elexacaftor, tezacaftor, and ivacaftor, for oral treatment of cystic fibrosis (CF) in patients ≥12 years old who have at least one Phe508del mutation in the CFTR gene. About 90% of patients with CF have at least one copy of the Phe508del (also called F508del) mutation. This is the first approval for elexacaftor. Ivacaftor is available alone (Kalydeco) and in 2-drug combinations with tezacaftor (Symdeco) and lumacaftor...

The FDA has approved ivabradine (Corlanor – Amgen) to reduce the risk of hospitalization for worsening heart failure in adults with stable, symptomatic chronic heart failure with left ventricular ejection fraction (LVEF) ≤35% who are in sinus rhythm with a resting heart rate ≥70 beats per minute and who are on maximum tolerated doses of beta blockers or have a contraindication to beta blocker use. Ivabradine has been available internationally for years as Procoralan and Corlentor for treatment of stable angina and heart failure.

The FDA has approved lixisenatide (Sanofi), a short-acting injectable GLP-1 (glucagon-like peptide-1) receptor agonist, for once-daily treatment of adults with type 2 diabetes, both alone (Adlyxin) and in a fixed-ratio combination with insulin glargine (Soliqua 100/33). Lixisenatide has been available since 2013 in many other countries as Lyxumia. It is the fifth GLP-1 receptor agonist to be approved in the US.

Changes caused by one drug in the absorption, distribution, metabolism or excretion of another may lead to a pharmacokinetic adverse drug interaction (DN Juurlink et al, JAMA 2003; 289:1652). Additive drug interactions, such as vasodilation caused by both sildenafil (Viagra) and nitrates, can also have adverse effects.

The FDA has approved the interleukin (IL)-6 inhibitor sarilumab (Kevzara – Sanofi) for second-line treatment of adults with moderately to severely active rheumatoid arthritis (RA). It is the second IL-6 inhibitor to be approved for this indication; tocilizumab (Actemra) was approved earlier.

Bipolar disorder is characterized by episodes of mania, hypomania, and depression. Recurrences of manic or (more frequently) depressive symptoms are common. About 15-20% of patients with bipolar disorder die by suicide.

About 2.5 million persons in the US had hepatitis C between 2017 and 2020. Guidelines from the American Association for the Study of Liver Diseases (AASLD) and the Infectious Diseases Society of America (IDSA) on management of hepatitis C virus (HCV) infection were updated in 2023.

Among patients with chronic heart failure, those with a left ventricular ejection fraction (LVEF) ≤40% are considered to have heart failure with reduced ejection fraction (HFrEF). Patients with a LVEF ≥50% are considered to have heart failure with preserved ejection fraction (HFpEF). Those with a LVEF of 41-49% are an intermediate group more similar to patients with HFpEF.

The subcutaneously injected interleukin-13 (IL-13) antagonist tralokinumab-ldrm (Adbry – Leo) and the oral Janus kinase (JAK) inhibitors abrocitinib (Cibinqo – Pfizer) and upadacitinib (Rinvoq – Abbvie) have been approved by the FDA for treatment of moderate to severe atopic dermatitis.

The FDA has approved nivolumab (Opdivo – BMS), an IV programmed death receptor-1 (PD-1) blocking antibody, for treatment of unresectable or metastatic melanoma that has progressed following treatment with ipilimumab (and a BRAF inhibitor in patients who are BRAF V600 mutation positive) and for treatment of metastatic squamous non-small cell lung cancer (NSCLC) that has progressed on or after platinum-based chemotherapy. It is the second PD-1 inhibitor to be marketed in the US after pembrolizumab (Keytruda), and the first to be approved for treatment of NSCLC.