The FDA has approved pegaptanib sodium (Macugen - Pfizer/Eyetech) as an intravitreous injection for treatment of all subtypes of neovascular (wet) age-related macular degeneration (AMD). The only other approved treatment for this condition is photodynamic therapy with intravenous verteporfin (Visudyne), which is recommended only for patients with one subtype (predominantly classic) of neovascular AMD.

Sunitinib (Sutent - Pfizer) and sorafenib (Nexavar - Bayer), two oral tyrosine kinase inhibitors, have been approved by the FDA for treatment of advanced renal cell carcinoma (RCC). Sunitinib is also approved for use in patients with gastrointestinal stromal tumor (GIST) who are not responding to or are intolerant of standard therapy with imatinib (Gleevec).

Sodium phenylbutyrate, an "orphan drug,"has recently been marketed for the treatment of patients with urea cycle disorders caused by a deficiency in one of the following hepatic enzymes: carbamyl phosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS). Urea cycle disorders are rare; they occur in one of every 10,000 births.

The FDA recently approved vandetanib [van det´ a nib] (Caprelsa – AstraZeneca) for oral treatment of symptomatic or progressive medullary thyroid cancer (MTC) in patients with unresectable locally advanced or metastatic disease. Vandetanib is the first drug approved in the US for medullary thyroid cancer.

Ado-trastuzumab emtansine (Kadcyla – Genentech), a human epidermal growth factor receptor 2 (HER2)-targeted antibody and microtubule inhibitor conjugate, has been approved by the FDA for intravenous (IV) treatment of HER2-positive metastatic breast cancer in patients previously treated with trastuzumab (Herceptin) and a taxane. The prefix was added to the new conjugate’s name at the request of the FDA to distinguish it from trastuzumab.

The FDA has approved cemiplimab-rwlc (Libtayo – Regeneron), an immune checkpoint inhibitor, for use in combination with platinum-based chemotherapy for first-line treatment of locally advanced or metastatic non-small cell lung cancer (NSCLC) in adults with no epidermal growth factor receptor (EGFR), anaplastic lymphoma kinase (ALK), or ROS1 aberrations and who are not candidates for surgical resection or chemoradiation. The drug was previously approved for first-line treatment of NSCLC in patients whose tumors have high PD-L1 expression and no genomic tumor aberrations....

A drug that combines the neutral sulfate salts of dextroamphetamine and racemic amphetamine with the dextro isomer of amphetamine saccharate and d,l amphetamine aspartate (Adderall - Richwood Pharmaceutical) has recently been promoted for treatment of children with Attention-Deficit/Hyperactivity Disorder (ADHD). This same mixture of amphetamine salts was previously marketed as Obetrol for treatment of obesity.

An intranasal spray formulation of the topical corticosteroid mometasone furoate, marketed for treatment of seasonal and perennial allergic rhinitis, has been advertised on television directly to the public.

Eflornithine hydrochloride cream 13.9% (Vaniqa - Bristol-Myers Squibb) has been approved by the FDA for reduction of unwanted facial hair in women.

The FDA has approved the oral kinase inhibitor encorafenib (Braftovi – Pfizer), in combination with the epidermal growth factor receptor (EGFR) inhibitor cetuximab (Erbitux), for treatment of adults with metastatic colorectal cancer (CRC) with a BRAF V600E mutation. Encorafenib was approved in 2018 for use with the mitogen-activated kinase (MEK) inhibitor binimetinib (Mektovi) for treatment of unresectable or metastatic melanoma with a BRAF V600E or V600K mutation.