The oral tyrosine kinase inhibitor neratinib (Nerlynx – Puma Biotechnology) has been approved by the FDA for use in combination with capecitabine (Xeloda, and generics) for treatment of advanced or metastatic human epidermal growth factor receptor 2 (HER2)-positive breast cancer in adults who received ≥2 prior anti-HER2-based regimens for metastatic disease. It was previously approved for use as monotherapy for extended adjuvant treatment of adults with early-stage, HER2-positive breast cancer following adjuvant trastuzumab (Herceptin)-based therapy. HER2 is overexpressed in...

The oral kinase inhibitor tucatinib (Tukysa – Seagen) has received accelerated approval from the FDA for use in combination with trastuzumab (Herceptin) for treatment of adults with RAS wild-type human epidermal growth factor receptor 2 (HER2)-positive unresectable or metastatic colorectal cancer that has progressed following treatment with fluoropyrimidine-, oxaliplatin-, and irinotecan-based chemotherapy regimens. Tucatinib was approved in 2020 for use in combination with trastuzumab and capecitabine (Xeloda, and generics) for treatment of adults with advanced unresectable or...

The FDA has approved the pyrimidine analog uridine triacetate (Xuriden [zur' uh den] – Wellstat Therapeutics) for treatment of hereditary orotic aciduria, a rare autosomal recessive disorder (estimated birth prevalence: <1:1,000,000) in infants and children caused by a deficiency in uridine 5'-monophosphate (UMP) synthase. This deficiency prevents synthesis of uridine nucleotides and causes developmental delays, failure to gain weight, hematologic abnormalities, and excessive urinary excretion of orotic acid, which can lead to urinary obstruction.1 Xuriden is the first drug to be approved...

With the 2004 Olympics only weeks away, performance-enhancing drugs will once again be receiving a great deal of attention. The US Anti-Doping Agency has published a list of drugs banned in Olympic sports (www.usantidoping.org) that includes, at least for some sports, all but 2 of the drugs reviewed here.

The FDA has approved teduglutide (te due’ gloo tide; Gattex – NPS), a recombinant DNA analog of glucagon-like peptide-2 (GLP-2), for treatment of short bowel syndrome (SBS) in adults who are dependent on parenteral support.

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.

Lazertinib (Lazcluze – Janssen Biotech), an oral kinase inhibitor, has been approved by the FDA for use in combination with the EGFR-MET bispecific antibody amivantamab (Rybrevant) for first-line treatment of locally advanced or metastatic non-small cell lung cancer (NSCLC) in adults with epidermal growth factor receptor (EGFR) exon 19 deletions or exon 21 L858R substitution mutations. This is the first approval for Lazcluze; amivantamab was previously approved for use alone and in combination with carboplatin and pemetrexed for treatment of NSCLC with EGFR exon 20 insertion...

Vascular endothelial growth factor (VEGF) is a principal mediator of neovascularization in wet age-related macular degeneration (AMD) and diabetic macular edema. It induces angiogenesis and increases vascular permeability and inflammation. VEGF inhibitors reduce leakage from blood vessels, prevent proliferation of new abnormal vessels, decrease swelling of the retina, and improve visual acuity in patients with neovascular (wet) AMD and diabetic macular edema. These drugs are given as periodic intravitreal injections with topical anesthesia.

Palbociclib (Ibrance – Pfizer), an oral cyclin-dependent kinase inhibitor, has been approved by the FDA for use in combination with the aromatase inhibitor letrozole (Femara, and generics) for first-line treatment of postmenopausal women with estrogen receptor (ER)-positive, human epidermal growth factor receptor 2 (HER2)-negative metastatic breast cancer. It is the first cyclin-dependent kinase inhibitor to become available in the US.

Teprotumumab-trbw (Tepezza - Horizon), an insulin-like growth factor-1 receptor (IGF-1R) inhibitor, has been approved by the FDA for IV treatment of thyroid eye disease. It is the first drug to be approved in the US for this indication.