Azacitidine (Vidaza - Pharmion), a pyrimidine nucleoside analog of cytidine, is the first drug approved by the FDA for treatment of myelodysplastic syndrome (MDS). Azacitidine is incorporated into newly synthesized DNA and inhibits DNA methyltransferase. Hypomethylation of DNA can restore the normal expression of genes critical for cell differentiation.

Duetact (Takeda), a new fixed-dose combination of the thiazolidinedione pioglitazone (Actos) and the sulfonylurea glimepiride (Amaryl, and others), was recently approved by the FDA for treatment of type 2 diabetes. It is approved as an adjunct to diet and exercise to improve glycemic control in patients already taking pioglitazone and/or a sulfonylurea. Avandaryl, a combination of glimepiride and the thiazolidinedione rosiglitazone, was approved in 2006.

Vorinostat (Zolinza - Merck), an oral histone deacetylase (HDAC) inhibitor, has received accelerated approval from the FDA for treatment of skin manifestations of cutaneous T-cell lymphoma (CTCL) that is persistent, progressive or recurrent after two systemic therapies. The most common types of CTCL are mycosis fungoides and SΘzary syndrome, the leukemic form of mycosis fungoides.

The FDA has approved ThermaClear (Therative), a battery-powered, handheld device, to treat individual acne lesions with heat. ThermaClear is indicated only for use on mild to moderate inflammatory acne, not severe nodular or severe cystic acne, and it is not meant to be used on blackheads and whiteheads. Two similar devices are already on the market: Zeno, another handheld device that delivers heat to acne lesions, and the Radiancy Clear Touch Lite Acne Clearance System, a larger heat-delivery device.

Treatment options for snoring and obstructive sleep apnea (OSA) now include, in addition to lifestyle remedies such as weight loss and avoidance of alcohol in the evening, mechanical devices, injection of botulinum toxin into the soft palate and a variety of ENT surgical outpatient procedures. Palatal implants (Pillar procedure - Restore Medical Inc) have been approved by the FDA for treatment of snoring and OSA.

The FDA has approved ezogabine (ee-ZOE-ga-been; Potiga – GSK/Valeant) for oral adjunctive treatment of partial-onset seizures in adults. Ezogabine is available in Europe as retigabine (Trobalt).

Most colorectal cancers arise from localized adenomatous polyps in a process that may take 10 years or more. Early detection and removal of a precancerous colonic lesion (polyp) can prevent cancer from developing.

The FDA has approved inclisiran (Leqvio – Novartis), a small interfering RNA (siRNA) directed to proprotein convertase subtilisin/kexin type 9 (PCSK9) mRNA, as an adjunct to diet and maximally tolerated statin therapy for subcutaneous (SC) treatment of adults with heterozygous familial hypercholesterolemia (HeFH) or clinical atherosclerotic cardiovascular disease (ASCVD) who require additional lowering of low-density lipoprotein cholesterol (LDL-C). Inclisiran is the first FDA-approved PCSK9-directed siRNA therapeutic agent.

The FDA has approved the hypoxia-inducible factor prolyl hydroxylase inhibitor (HIF-PHI) vadadustat (Vafseo – Akebia) for oral treatment of anemia due to chronic kidney disease (CKD) in adults who have been on dialysis for at least 3 months. Vadadustat is the second HIF-PHI to be approved in the US for this indication; daprodustat (Jesduvroq) was approved earlier, but it was withdrawn from the US market in 2024 for commercial reasons.

Tuberculosis (TB) is still a common cause of death worldwide, and the prevalence of drug-resistant TB poses challenges to its treatment and control. Guidelines with detailed management recommendations are available from the American Thoracic Society, Centers for Disease Control and Prevention (CDC) and Infectious Diseases Society of America (IDSA).