The FDA has approved a new low-dose formulation of the tricyclic antidepressant doxepin (Silenor – Somaxon) for treatment of insomnia associated with sleep maintenance. The manufacturer claims that this dose retains the hypnotic effect of doxepin, without typical tricyclic adverse effects. Doxepin is available generically in higher-strength capsules and in a liquid formulation.

The FDA has approved an oral liquid formulation of glycerol phenylbutyrate (Ravicti – Hyperion) for chronic management of patients ≥2 years old with urea cycle disorders that cannot be adequately managed by a protein- restricted diet. Sodium phenylbutyrate (Buphenyl, and generics), another oral drug approved by the FDA for this indication, has a bitter taste. The new product, which contains no sodium, has little or no taste. Either drug must be used in addition to a protein-restricted diet and, if needed, dietary supplementation with amino acids and other nutrients.

The FDA has approved the oral tyrosine kinase inhibitor avapritinib (Ayvakit – Blueprint Medicines) for treatment of unresectable or metastatic gastrointestinal stromal tumors (GISTs) harboring a platelet-derived growth factor receptor alpha (PDGFRA) D842V or other PDGFRA exon 18 mutation. Avapritinib is the first drug to be approved for this indication in the US.

The FDA has approved the partial opioid agonist buprenorphine in a transdermal formulation (Butrans – Purdue) for treatment of moderate to severe chronic pain. Buprenorphine has been available in the US for years in parenteral formulations for pain and in sublingual tablets for opioid dependence.1 Transdermal buprenorphine has been available in Europe for several years.2

An intranasal formulation of ketorolac tromethamine (Sprix – Lutipold) has been approved by the FDA for short-term (up to 5 days) treatment of moderate to moderately severe pain in adults. It is the first nonsteroidal anti-inflammatory drug (NSAID) to become available in an intranasal formulation. Ketorolac tromethamine is also available in oral, ophthalmic and injectable formulations.

The oral kinase inhibitor futibatinib (Lytgobi – Taiho) has received accelerated approval from the FDA for treatment of adults with previously treated, unresectable, locally advanced or metastatic intrahepatic cholangiocarcinoma with fibroblast growth factor receptor 2 (FGFR2) fusions or other rearrangements. Accelerated approval was based on the overall response rate and duration of response. Futibatinib is the second drug to be approved for this indication; pemigatinib (Pemazyre) was approved earlier.

Tisotumab vedotin-tftv (Tivdak – Seagen/Genmab), a tissue factor-directed antibody and microtubule inhibitor conjugate, has received full approval from the FDA for treatment of recurrent or metastatic cervical cancer that progressed on or after chemotherapy. It is the first antibody-drug conjugate to be approved for treatment of cervical cancer. The drug received accelerated approval from the FDA in 2021 for the same indication.

The goal for drug therapy of type 2 diabetes is achieving and maintaining a near-normal glycated hemoglobin (HbA1C) concentration without inducing hypoglycemia; the target has generally been an HbA1C of 6.5-7.0% or lower. Whether treating to this level prevents macrovascular (cardiovascular) events has been unclear. Now, 2 large randomized, double-blind trials in patients with long-standing diabetes and at high risk for cardiovascular disease have found no decrease in macrovascular events with intensive glucose control.The ACCORD trial in about 10,000 patients found that patients intensively...

The FDA has approved a once-daily extended-release (ER) formulation of the antiepileptic topiramate (Trokendi XR – Supernus) for initial monotherapy in patients ≥10 years old with partial onset seizures or primary generalized tonic-clonic seizures and for adjunctive therapy in patients ≥6 years old with partial onset seizures, primary generalized tonic-clonic seizures, or seizures associated with Lennox-Gastaut syndrome. Topiramate has been available for many years as an immediate-release (IR) formulation (Topamax, and generics) for the same indications in patients ≥2...

Difelikefalin (Korsuva – Vifor), an IV kappa opioid receptor (KOR) agonist, has been approved by the FDA for treatment of moderate to severe pruritus associated with chronic kidney disease (CKD) in adults on hemodialysis. It is the first drug to be approved for this indication and the first KOR agonist to become available in the US. Difelikefalin has not been studied in patients on peritoneal dialysis.