Lenalidomide (Revlimid - Celgene), a thalidomide analog, has been approved by the FDA for treatment of patients with transfusion-dependent anemia due to myelodysplastic syndrome (MDS) associated with a 5q deletion cytogenetic abnormality and a low or low-intermediate risk of mortality and progression to leukemia (based on the International Prognostic Scoring System).

Use of a selective serotonin reuptake inhibitor (SSRI) is common in women taking tamoxifen (Nolvadex, and others) for breast cancer, both to treat depression and to decrease hot flashes. However, tamoxifen must be metabolized by CYP2D6 to become pharmacologically fully active (MJ Higgins et al. J Natl Compr Canc Netw 2009; 7:203), and the SSRIs fluoxetine (Prozac, and others) and paroxetine (Paxil, and others) are strong inhibitors of CYP2D6. Sertraline (Zoloft, and others) inhibits CYP2D6 to a lesser extent. Citalopram (Celexa, and others) and escitalopram (Lexapro), the 2 other SSRIs...

The FDA has approved velaglucerase alfa (Vpriv – Shire), a new formulation of glucocerebrosidase prepared from human fibroblasts, for treatment of the nonneurologic form of Gaucher’s disease (Type 1). Patients with Gaucher’s disease have a congenital deficiency of glucocerebrosidase that leads to accumulation of glucosylceramide, the end-product of sphingolipid catabolism, in the lysozymes of reticuloendothelial cells in the liver, spleen and bone marrow.Velaglucerase is the second form of the enzyme now available in the US; imiglucerase (Cerezyme – Genzyme), which is produced by...

The FDA has approved droxidopa (Northera – Lundbeck) for oral treatment of adults with symptomatic neurogenic orthostatic hypotension (NOH) caused by primary autonomic failure (Parkinson's disease, multiple system atrophy, or pure autonomic failure), dopamine beta-hydroxylase deficiency, or nondiabetic autonomic neuropathy. This is the first approval for droxidopa in the US. It has been available in Japan for use in NOH since 1989.

The FDA has approved Liletta, an intrauterine device (IUD) that releases the synthetic progestin levonorgestrel, for prevention of pregnancy for up to 3 years. The fourth IUD to be approved in the US, and the third that releases levonorgestrel, Liletta is comarketed by Actavis and Medicines360, a nonprofit women's health pharmaceutical company. The wholesale cost of Liletta is similar to that of other IUDs (see Table 1), but the manufacturer offers programs that significantly reduce the cost for commercially-insured patients and clinics serving low-income women.

The FDA has approved Vuity (Abbvie), a 1.25% ophthalmic solution of the muscarinic receptor agonist pilocarpine hydrochloride, for treatment of presbyopia in adults. Pilocarpine 1%, 2%, and 4% ophthalmic solutions (Isopto Carpine, and others) have been available for years for treatment of glaucoma, but local and systemic adverse effects have limited their use.

Fam-trastuzumab deruxtecan-nxki (Enhertu – Daiichi Sankyo/AstraZeneca), which received accelerated approval by the FDA in 2019 for treatment of HER2-positive breast cancer based on its rate and duration of response,1 has been granted regular approval for treatment of adults with unresectable or metastatic HER2-positive breast cancer who received a prior anti-HER2-based regimen in the metastatic setting or in the neoadjuvant or adjuvant setting and developed recurrence during or within 6 months of completing treatment.

Erdafitinib (Balversa – Janssen), an oral kinase inhibitor, has received full approval from the FDA for treatment of locally advanced or metastatic urothelial carcinoma in adults with susceptible FGFR3 (fibroblast growth factor receptor) genetic alterations who had disease progression on or after at least one prior line of systemic therapy. It is not recommended for use in patients who are eligible for but have not received prior PD-1 (programmed death receptor-1) or PD-L1 (programmed death-ligand 1) inhibitor therapy. Erdafitinib is the first oral FGFR kinase inhibitor to be...

The FDA has approved tebentafusp-tebn (Kimmtrak – Immunocore), a first-in-class bispecific gp100 peptide-HLA-directed CD3 T-cell engager, for treatment of HLA-A*02:01-positive unresectable or metastatic uveal melanoma in adults.

Tarlatamab-dlle (Imdelltra – Amgen), a first-in-class bispecific delta-like ligand 3 (DLL3)-directed CD3 T-cell engager, has received accelerated approval from the FDA for treatment of extensive-stage small cell lung cancer (SCLC) in adults who had disease progression on or after platinum-based chemotherapy. It is the first bispecific DLL3-directed CD3 T-cell engager to be approved in the US for this indication. Most patients with SCLC have a response to initial treatment, but progression generally occurs within a few months and overall survival is usually less than 8 months....