The FDA has approved Cobenfy (BMS), an oral fixed-dose combination of the muscarinic agonist xanomeline and the peripheral muscarinic antagonist trospium chloride, for treatment of schizophrenia in adults. It is the first antipsychotic drug to be approved in the US for treatment of schizophrenia that does not block dopamine receptors. This is the first approval for xanomeline; trospium has been available for many years for treatment of overactive bladder.

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.

The FDA has approved Zunveyl (Alpha Cognition), a delayed-release formulation of the prodrug benzgalantamine, for treatment of mild to moderate dementia of Alzheimer's disease (AD). Immediate- and extended-release formulations of the acetylcholinesterase inhibitor galantamine have been available in the US for years for treatment of AD.

The FDA has approved vonoprazan, a potassium-competitive acid blocker, copackaged with amoxicillin (Voquezna Dual Pak – Phathom) and with amoxicillin and clarithromycin (Voquezna Triple Pak) for treatment of Helicobacter pylori infection in adults. Vonoprazan is the first potassium-competitive acid blocker to be approved in the US. It has been available in Japan for treatment of various acid-related disorders since 2014. Vonoprazan is not available alone in the US.

The FDA has granted regular approval to the oral kinase inhibitor tepotinib (Tepmetko – EMD Serono) for treatment of adults with metastatic non-small cell lung cancer (NSCLC) harboring mesenchymal-epithelial transition (MET) exon 14 skipping alterations. MET exon 14 skipping mutations occur in 3-4% of NSCLC cases and are associated with advanced disease and a poor prognosis. Tepotinib received accelerated approval for the same indication in 2021 based on initial overall response rates and duration of response.

The FDA has approved a combination oral contraceptive containing the estrogen estetrol and the progestin drospirenone (Nextstellis – Mithra/Mayne). Estetrol is the first new estrogen to become available in the US in 50 years. Drospirenone is available alone (Slynd) and in combinations with the estrogen ethinyl estradiol for prevention of pregnancy.

The FDA has approved the oral second-generation antipsychotic drug brexpiprazole (Rexulti – Otsuka/Lundbeck) for once-daily treatment of agitation associated with dementia due to Alzheimer's disease (AD). Brexpiprazole is the first drug to be approved in the US for this indication. It is also approved for treatment of schizophrenia and as an adjunct to antidepressants for treatment of major depressive disorder.

Elafibranor (Iqirvo – Ipsen), a peroxisome proliferator-activated receptor (PPAR) agonist, has been granted accelerated approval by the FDA for treatment of primary biliary cholangitis (PBC) in adults. It is indicated for use in combination with ursodeoxycholic acid (ursodiol, UDCA; Urso, and others) in patients with an inadequate response to UDCA alone and as monotherapy in those unable to tolerate UDCA.

View the Expanded Table: Some Oral Drugs for Chronic Insomnia

A once-weekly transdermal formulation of the acetylcholinesterase inhibitor donepezil (Adlarity – Corium) has been approved by the FDA for treatment of mild, moderate, and severe Alzheimer's disease (AD) dementia. Donepezil is the second acetylcholinesterase inhibitor to become available in a transdermal formulation; transdermal rivastigmine (Exelon Patch), which is applied once daily, has been available for years for the same indication. Donepezil is also available in oral formulations (Aricept, and generics) for treatment of AD dementia.