The FDA has approved the oral tyrosine kinase inhibitor ripretinib (Qinlock – Deciphera) for treatment of adults with advanced gastrointestinal stromal tumors (GISTs) who have previously received treatment with ≥3 kinase inhibitors, including imatinib (Gleevec, and generics).

The FDA has approved the selective beta-3 adrenergic agonist vibegron (Gemtesa – Urovant Sciences) for treatment of overactive bladder in adults with symptoms of urge urinary incontinence, urgency, and urinary frequency. It is the second beta-3 agonist to be approved in the US; mirabegron (Myrbetriq) was the first.

Tirbanibulin, a microtubule inhibitor, has been approved by the FDA as a 1% ointment (Klisyri – Almirall) for topical treatment of actinic keratosis of the face or scalp.

Berotralstat (Orladeyo – Biocryst), an oral plasma kallikrein inhibitor, has been approved by the FDA for prevention of hereditary angioedema (HAE) attacks in adults and children ≥12 years old. The subcutaneously-injected plasma kallikrein inhibitor lanadelumab-flyo (Takhzyro) and the human plasma-derived C1 esterase inhibitors (C1INHs) Cinryze, which is given IV, and Haegarda, which is given SC, have been available for prophylaxis of HAE for years.

The oral second-generation antipsychotic drug lumateperone (Caplyta – Intra-Cellular Therapies), which was approved by the FDA in 2020 for treatment of schizophrenia, is now approved for use as monotherapy or as an adjunct to lithium or valproate for treatment of depressive episodes associated with bipolar I or II disorder in adults.

The FDA has approved Entadfi (Veru), a fixed-dose combination of the 5α-reductase inhibitor finasteride and the phosphodiesterase type 5 (PDE5) inhibitor tadalafil, for initial treatment of benign prostatic hyperplasia (BPH) in men with an enlarged prostate; use of the drug is limited to 26 weeks. Entadfi is the first 5α-reductase inhibitor/PDE5 inhibitor combination to become available in the US. Finasteride (Proscar, and generics) and tadalafil (Cialis, and generics) are also available separately.

The recombinant interleukin-1 (IL-1) receptor antagonist anakinra (Kineret – Sobi) has been granted an FDA Emergency Use Authorization (EUA) for treatment of hospitalized adults with confirmed COVID-19 pneumonia who require low- or high-flow supplemental oxygen, are at risk of progressing to severe respiratory failure, and are likely to have elevated plasma levels of soluble urokinase plasminogen activator receptor (suPAR). Anakinra has been available in the US for years; it is FDA-approved for multiple indications, including rheumatoid arthritis. Assays for suPAR are...

The FDA has approved Vowst (Seres Therapeutics/Nestle HealthScience), an oral capsule containing live fecal microbiota spores, for prevention of additional recurrences of Clostridioides difficile infection (CDI) in adults. Vowst is the first orally administered microbiota-based treatment to be approved for this indication. A rectally-administered live fecal microbiota-based suspension (Rebyota) was approved in 2022 for the same indication. Neither product is approved for acute treatment of CDI.

The FDA has approved the interleukin (IL)-6 inhibitor sarilumab (Kevzara – Sanofi/Regeneron) for treatment of polymyalgia rheumatica (PMR) in adults who had an inadequate response to corticosteroids or cannot tolerate a corticosteroid taper. Sarilumab is the first biologic drug to be approved for treatment of PMR; it was previously approved for treatment of rheumatoid arthritis.

The FDA has approved an epinephrine nasal spray (neffy – ARS Pharma) for emergency treatment of type 1 hypersensitivity reactions including anaphylaxis in patients who weigh ≥30 kg. It is the first noninjectable epinephrine product to be approved for this indication.