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.

Betibeglogene autotemcel (Zynteglo — Bluebird Bio), an autologous lentiviral vector cell-based gene therapy, has been approved by the FDA for one-time treatment of transfusion-dependent beta thalassemia in children and adults. Exagamglogene autotemcel (Casgevy), a cell-based gene therapy that uses CRISPR/Cas9 gene-editing technology, was approved earlier this year for the same indication in patients ≥12 years old.

The FDA has approved imetelstat (Rytelo – Geron), a first-in-class telomerase inhibitor, for treatment of low- to intermediate-1 risk myelodysplastic syndromes (MDS) in adults with transfusion-dependent anemia requiring 4 or more red blood cell (RBC) units over 8 weeks who have not responded to, are no longer responding to, or are ineligible for erythropoiesis-stimulating agents (ESAs).

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...

Mitoxantrone (Novantrone - Lederle), a synthetic anthracene related to the anthracyclines doxorubicin (Adriamycin) and daunorubicin (Cerubidine), has now been marketed in the USA to be used in combination with other drugs for initial treatment of acute nonlymphocytic leukemia in adults.

The FDA has approved dimethyl fumarate (Tecfidera – Biogen Idec), formerly called BG-12, for treatment of relapsing forms of multiple sclerosis (MS). It is the third oral drug to be approved in recent years for this indication.

Trastuzumab (Herceptin - Genentech), a recombinant 'humanized' monoclonal antibody (rhuMAb) that binds to a protein encoded by the oncogene HER2, and capecitabine (ka pe site' a been; Xeloda - Roche), an oral pro-drug for 5-fluorouracil, have been approved by the FDA for treatment of metastatic breast cancer.

The FDA has approved the oral tyrosine kinase inhibitor ripretinib (Qinlock – Deciphera) for treatment of adults with advanced gastrointestinal stromal tumors (GISTs) who have previously received treatment with ≥3 kinase inhibitors, including imatinib (Gleevec, and generics).

The oral retinoid isotretinoin (Accutane, and others) is the most effective drug available for treatment of acne.1 The FDA has recently approved a new oral isotretinoin product (Absorica – Cipher/Ranbaxy) for treatment of severe, recalcitrant nodular acne.Isotretinoin can completely clear severe nodulocystic lesions, in some cases leading to remission that can persist for years after treatment is stopped. Mucocutaneous adverse effects include dry skin, dry eye, epistaxis, nasopharyngitis, cheilitis, alopecia, eczema, skin fragility and photosensitivity. Musculoskeletal symptoms,...

The androgen receptor inhibitor darolutamide (Nubeqa – Bayer) has been approved by the FDA for use in combination with docetaxel for treatment of metastatic hormone-sensitive prostate cancer (mHSPC). The drug was previously approved for treatment of nonmetastatic castration-resistant prostate cancer (nmCRPC).