The FDA has approved telotristat ethyl (Xermelo – Lexicon), a tryptophan hydroxylase inhibitor, for use in combination with a somatostatin analog (SSA) for treatment of carcinoid syndrome diarrhea inadequately controlled with SSA therapy alone. Telotristat ethyl is the first oral drug to be approved in the US for this indication.

The FDA has approved the interleukin-6 (IL-6) receptor antagonist tocilizumab (Actemra – Genentech) for subcutaneous (SC) treatment of giant cell arteritis in adults. It is the first drug to be approved in the US for this indication. Tocilizumab is also approved for treatment of rheumatoid arthritis, polyarticular or systemic juvenile idiopathic arthritis, and cytokine release syndrome.

The FDA has approved lutetium Lu 177 dotatate (Lutathera – Advanced Accelerator Applications), a radiolabeled somatostatin analog, for treatment of somatostatin receptor-positive gastroenteropancreatic neuroendocrine tumors (GEPNETs) in adults.

The FDA has approved Semglee (Mylan), an insulin glargine product similar to Lantus, for treatment of type 1 diabetes in children and adults and type 2 diabetes in adults. Semglee is the second "follow-on" insulin glargine product to become available in the US; Basaglar, which is also similar to Lantus, was the first. Lantus is a recombinant analog of human insulin that forms microprecipitates in subcutaneous tissue, prolonging its duration of action to a mean of about 24 hours with no pronounced peak effect.

The FDA has approved lanadelumab-flyo (Takhzyro – Takeda), a subcutaneously-administered plasma kallikrein inhibitor, for prevention of hereditary angioedema (HAE) attacks in adults and children ≥12 years old. Lanadelumab is the third drug to be approved for this indication; the human plasmaderived C1 esterase inhibitors (C1INHs) Cinryze, which is administered IV, and Haegarda, which is given SC, have been available for years for HAE prophylaxis in patients ≥6 years old.

The opioid agonist tramadol is now available in an oral solution (Qdolo – Athena Bioscience) for management of pain severe enough to require an opioid and for which alternative treatment options are inadequate. Tramadol has been available for years in immediate-release tablets and capsules, extended-release capsules (Ultram, and others), and in a fixed-dose combination tablet with acetaminophen (Ultracet, and generics).

The FDA has approved setmelanotide (Imcivree – Rhythm), a subcutaneously injected melanocortin 4 (MC4) receptor agonist, for chronic weight management in patients ≥6 years old with obesity due to pro-opiomelanocortin (POMC) deficiency, proprotein subtilisin/kexin type 1 (PCSK1) deficiency, or leptin receptor (LEPR) deficiency.

Rilonacept (Arcalyst – Kiniksa), an interleukin-1 (IL-1) antagonist that has been available for years for treatment of cryopyrin-associated periodic syndromes, has now been approved by the FDA for treatment of recurrent pericarditis and prevention of further recurrences in patients ≥12 years old. It is the first drug to be approved in the US for this indication. Anakinra (Kineret), an IL-1 receptor antagonist FDA-approved for treatment of rheumatoid arthritis, has been used off-label for years for treatment of recurrent pericarditis.

The FDA has approved an over-the-counter (OTC) liquid-filled capsule formulation of aspirin (Vazalore – PLx Pharma). The manufacturer has been heavily promoting Vazalore with claims of fast, predictable absorption and antiplatelet activity and improved gastrointestinal safety compared to existing OTC aspirin formulations.

The oral kinase inhibitor alpelisib (Vijoice – Novartis) has been approved by the FDA for treatment of patients ≥2 years old with severe manifestations of PIK3CA-related overgrowth spectrum (PROS) who require systemic treatment. Alpelisib is the first drug to be approved in the US for this indication. It was also approved in 2019 as Piqray for use in combination with fulvestrant for treatment of certain types of breast cancer.