Thalidomide is now available as an investigational drug in the USA. A synthetic derivative of glutamic acid, it was marketed in Europe in 1957 as a sedative but withdrawn four years later after being associated with severe human teratogenicity (PF DArcy and JP Griffin, Adverse Drug React Toxicol Rev, 13:65, 1994). The drug has since been found effective for several different indications.

The media recently reported that the FDA has threatened to withdraw Synthroid from the market, alarming many physicians and patients.

Isotretinoin (Accutane - Roche), an effective oral drug for treatment of acne, will soon be available generically. Concerns about its adverse effects, particularly psychiatric symptoms in adolescents, have been widely reported in the media.

Recombinant human α-L-iduronidase (laronidase; Aldurazyme BioMarin/Genzyme), an orphan drug, has been approved by the FDA for enzyme replacement therapy in mucopolysaccharidosis (MPS) type I, a lysosomal storage disorder caused by deficiency of the enzyme. This review describes the disease, treatment, adverse effects and cost.

An extended-release oral formulation of guanfacine hydrochloride (Intuniv – Shire), a selective alpha_2A-adrenergic agonist, has been approved by the FDA for treatment of attention-deficit/hyperactivity disorder (ADHD) in children 6-17 years old.

The FDA has approved the use of nitroglycerin ointment 0.4% (Rectiv – ProStrakan/Aptalis) for treatment of moderate to severe pain associated with chronic anal fissure; the same drug is marketed as Rectogesic in Europe. Nitroglycerin ointment 2% (Nitro-Bid) is available in the US for prevention of angina.

The FDA has approved sebelipase alfa (Kanuma – Alexion), a recombinant form of lysosomal acid lipase (LAL), for enzyme replacement therapy in patients with LAL deficiency. Sebelipase alfa is the first drug to be approved in the US for treatment of LAL deficiency, a rare autosomal recessive storage disease.

Xywav (Jazz), an oral solution that contains calcium, magnesium, potassium, and sodium oxybates, has been approved by the FDA for treatment of idiopathic hypersomnia in adults. It is the first drug to be approved in the US for this indication. Xywav was approved in 2020 for treatment of excessive daytime sleepiness or cataplexy in patients ≥7 years old with narcolepsy. It contains about 92% less sodium than sodium oxybate oral solution (Xyrem), which has been available in the US for years for use in patients ≥7 years old with narcolepsy.

The FDA has approved Acuvue Theravision with Ketotifen (Johnson & Johnson Vision Care), a daily-wear, disposable, vision-correcting soft contact lens that releases the H₁-antihistamine ketotifen, for prevention of ocular itch due to allergic conjunctivitis in contact lens users. This is the first approval for a drug-eluting contact lens. Ketotifen eye drops (Alaway, Zaditor, and others) are available over the counter.

Atezolizumab (Tecentriq – Genentech), an immune checkpoint inhibitor, has been approved by the FDA for treatment of unresectable or metastatic alveolar soft part sarcoma (ASPS) in patients ≥2 years old. It was previously approved for treatment of non-small cell lung cancer, small cell lung cancer, hepatocellular cancer, and melanoma (see Table 1). Atezolizumab is the first drug to be approved in the US for treatment of ASPS. ASPS is a rare disorder that affects mostly adolescents and young adults; <1% of soft tissue sarcomas are ASPS.