The FDA has approved a 1% cream formulation of the antiparasitic drug ivermectin (Soolantra – Galderma) for topical treatment of inflammatory lesions of rosacea. Ivermectin is available in the US in tablets (Stromectol, and generics) for treatment of onchocerciasis and other worm infestations and as a 0.5% lotion (Sklice) for treatment of head lice.

Quzyttir (TerSera), an IV formulation of the second-generation H1-antihistamine cetirizine, has been approved by the FDA for treatment of acute urticaria in patients ≥6 months old. Oral formulations of cetirizine (Zyrtec, and others) have been for sale over the counter for years. Quzyttir is the first parenteral formulation of a second-generation H1-antihistamine to become available in the US; parenteral formulations of two first-generation H1-antihistamines, diphenhydramine (Benadryl, and others) and hydroxyzine (Vistaril, and others), have been available for many...

Golodirsen (Vyondys 53 — Sarepta), an antisense oligonucleotide, has received accelerated approval from the FDA for treatment of Duchenne muscular dystrophy (DMD) in the ~8% of patients who have mutations of the dystrophin gene that are amenable to exon 53 skipping. It is the first drug to be approved for this indication and the third to be approved for treatment of DMD; the antisense oligonucleotide eteplirsen (Exondys 51) and the oral corticosteroid deflazacort (Emflaza) were approved earlier.

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.

The FDA has approved the oral tyrosine kinase inhibitor avapritinib (Ayvakit – Blueprint Medicines) for treatment of unresectable or metastatic gastrointestinal stromal tumors (GISTs) harboring a platelet-derived growth factor receptor alpha (PDGFRA) D842V or other PDGFRA exon 18 mutation. Avapritinib is the first drug to be approved for this indication in the US.

Casimersen (Amondys 45 – Sarepta), an IV antisense oligonucleotide, has received accelerated approval from the FDA for treatment of Duchenne muscular dystrophy (DMD) in patients who have mutations of the dystrophin gene that are amenable to exon 45 skipping (DMD-45), which occur in ~8% of DMD cases. Casimersen is the first drug to be approved for this indication and the fifth to be approved for treatment of DMD; the IV antisense oligonucleotides eteplirsen (Exondys 51), golodirsen (Vyondys 53), and viltolarsen (Viltepso) and the oral corticosteroid deflazacort (Emflaza) were...

Aducanumab-avwa (Aduhelm – Biogen/Eisai), an IV amyloid beta-directed monoclonal antibody, has received accelerated approval from the FDA for treatment of Alzheimer's disease. The approval was based on the surrogate endpoint of reduction in amyloid beta plaques in the brain. The manufacturer is required to conduct an additional randomized controlled trial to establish that reducing amyloid beta plaques with aducanumab improves clinical outcomes in patients with Alzheimer's disease.

Efgartigimod alfa-fcab (Vyvgart – Argenx), a first-in-class neonatal Fc receptor (FcRn) antagonist, has been approved by the FDA for IV treatment of generalized myasthenia gravis in adults who are anti-acetylcholine receptor (AChR) antibody positive. The IV complement inhibitor eculizumab (Soliris) was approved for the same indication in 2017.

The Bruton's tyrosine kinase (BTK) inhibitor pirtobrutinib (Jaypirca – Lilly) has received accelerated approval from the FDA for treatment of relapsed or refractory mantle cell lymphoma (MCL) in adults who received ≥2 prior lines of systemic therapy, including a BTK inhibitor. Accelerated approval was based on the response rate. The BTK inhibitors ibrutinib (Imbruvica), acalabrutinib (Calquence), and zanubrutinib (Brukinsa) are also approved for treatment of MCL.

Since our initial review of the oral lipid-lowering adenosine triphosphate-citrate lyase (ACL) inhibitor bempedoic acid (Nexletol – Esperion) in 2020, cardiovascular outcomes data in statin-intolerant patients have become available.