Delandistrogene moxeparvovec-rokl (Elevidys – Sarepta), an adeno-associated virus (AAV) vector-based gene therapy, has received accelerated approval from the FDA for treatment of ambulatory children 4-5 years old with Duchenne muscular dystrophy (DMD) who have a confirmed mutation in the dystrophin gene. It is the first gene therapy to be approved in the US for treatment of DMD. Accelerated approval was based on expression of microdystrophin in skeletal muscle, a surrogate endpoint.

The oral nonergot dopamine agonist pramipexole (Mirapex ER, and generics), which is FDA-approved for treatment of Parkinson's disease and restless legs syndrome, has been used off-label in patients with treatment-resistant depression (TRD). A double-blind, placebo-controlled trial of pramipexole augmentation in patients with unipolar TRD was recently published.

The FDA has recommended additional MRI monitoring during initiation of treatment with the amyloid beta-directed monoclonal antibody lecanemab-irmb (Leqembi) and has approved a new subcutaneously injected formulation of the drug (Leqembi Iqlik) for maintenance treatment of Alzheimer's disease.

The goal of asthma treatment is to control symptoms, prevent exacerbations, and maintain normal lung function. Management of acute exacerbations in the emergency department is not discussed here.

Epcoritamab-bysp (Epkinly – Genmab), a bispecific CD20-directed CD3 T-cell engager, has received accelerated approval from the FDA for subcutaneous treatment of relapsed or refractory diffuse large B-cell lymphoma (DLBCL), not otherwise specified, including DLBCL arising from indolent lymphoma, and high-grade B-cell lymphoma after ≥2 lines of systemic therapy. Epcoritamab is the second T-cell-engaging bispecific antibody to become available in the US for treatment of non-Hodgkin's lymphoma; mosunetuzumab-axgb (Lunsumio) was recently approved for relapsed or refractory...

The guanylate cyclase-C receptor agonist linaclotide (Linzess – Abbvie/Ironwood) was first approved by the FDA in 2012 for treatment of chronic idiopathic constipation and irritable bowel syndrome with constipation in adults. The drug has now been approved for treatment of functional constipation in patients 6-17 years old. Linaclotide is the only drug to be approved in the US for treatment of functional constipation.

Axatilimab-csfr (Niktimvo – Incyte), a colony stimulating factor-1 receptor-blocking antibody, has been approved by the FDA for intravenous treatment of chronic graft-versus-host disease (cGVHD) after failure of at least 2 lines of systemic therapy in patients weighing ≥40 kg. Corticosteroids are generally the first-line treatment for cGVHD. The oral kinase inhibitors ibrutinib (Imbruvica), ruxolitinib (Jakafi), and belumosudil (Rezurock) are also approved for treatment of cGVHD. Extracorporeal photopheresis has been used off-label.

The FDA has approved the interleukin-2 (IL-2) receptor-directed fusion protein denileukin diftitox (Lymphir – Citius) for treatment of relapsed or refractory stage I-III cutaneous T-cell lymphoma in adults who received at least one prior systemic therapy. Lymphir is a reformulated, purified version of Ontak, which was voluntarily withdrawn from the market in 2014 because of manufacturing issues.

The FDA has approved Vybrique (IBSA), an oral film formulation of the phosphodiesterase type 5 (PDE5) inhibitor sildenafil, for treatment of erectile dysfunction (ED). Sildenafil oral tablets (Viagra, and generics) have been available since 1998. The manufacturer is promoting the new product as a "discreet, on-the-go" option that can be taken without water.

The FDA has expanded the Emergency Use Authorizations (EUAs) for the mRNA-based COVID-19 vaccines manufactured by Pfizer/BioNTech (Comirnaty) and Moderna (Spikevax) and the adenovirus-based vaccine manufactured by Johnson & Johnson/Janssen to include administration of a booster dose in select populations after primary immunization with either the same COVID-19 vaccine or a different one.