Belzutifan (Welireg – Merck), a first-in-class hypoxia-inducible factor inhibitor, has been approved by the FDA for treatment of advanced renal cell carcinoma (RCC) in adults who received prior treatment with a programmed death receptor-1 (PD-1) or programmed death-ligand 1 (PD-L1) inhibitor and a vascular endothelial growth factor tyrosine kinase inhibitor (VEGF-TKI). Belzutifan was previously approved for use in patients with von Hippel-Lindau disease.

Berdazimer 10.3% gel (Zelsuvmi – Ligand), a first-in-class nitric oxide-releasing agent, has been approved by the FDA for topical treatment of molluscum contagiosum in patients ≥1 year old. It is the second drug to become available in the US for treatment of molluscum contagiosum and the first that can be applied by the patient or caregiver at home; cantharidin 0.7% solution (Ycanth), which is applied by a healthcare professional, was approved earlier for use in patients ≥2 years old.

Tisotumab vedotin-tftv (Tivdak – Seagen/Genmab), a tissue factor-directed antibody and microtubule inhibitor conjugate, has received full approval from the FDA for treatment of recurrent or metastatic cervical cancer that progressed on or after chemotherapy. It is the first antibody-drug conjugate to be approved for treatment of cervical cancer. The drug received accelerated approval from the FDA in 2021 for the same indication.

View the Table: GLP-1 and GIP/GLP-1 Receptor Agonists for Type 2 Diabetes

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...

Revumenib (Revuforj – Syndax), an oral menin inhibitor, has been approved by the FDA for treatment of relapsed or refractory acute leukemia in patients ≥1 year old with a lysine methyltransferase 2A gene (KMT2A) translocation. It is the first menin inhibitor to be approved in the US.

Mirdametinib (Gomekli – SpringWorks Therapeutics), an oral kinase inhibitor, has been approved by the FDA for treatment of neurofibromatosis type 1 (NF1) in patients ≥2 years old who have symptomatic plexiform neurofibromas not amenable to complete resection. Mirdametinib is the second drug to be approved for this indication in the US and the first to be approved for use in adults. The oral kinase inhibitor selumetinib (Koselugo) was approved in 2020 for use in patients 2-17 years old.

The FDA has approved diazoxide choline extended-release tablets (Vykat XR – Soleno Therapeutics) for treatment of hyperphagia in patients ≥4 years old with Prader-Willi syndrome. Diazoxide choline is the first drug to be approved in the US for this indication. Diazoxide oral suspension (Proglycem) has been available for years for management of symptomatic hypoglycemia.

The oral kinase inhibitor pemigatinib (Pemazyre – Incyte) has received accelerated approval from the FDA for treatment of adults with previously treated, unresectable, locally advanced or metastatic cholangiocarcinoma with fibroblast growth factor receptor 2 (FGFR2) fusions or other rearrangements. Pemigatinib is the first drug to be approved in the US for this indication.

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.