Remestemcel-L (Ryoncil – Mesoblast), an allogeneic bone marrow-derived mesenchymal stromal cell therapy, has been approved by the FDA for treatment of steroid-refractory acute graft-versus-host disease (GVHD) in patients ≥2 months old. Steroid-refractory GVHD is associated with a poor prognosis, organ damage, and death, particularly in pediatric patients. Remestemcel-L is the first cellular therapy to be approved in the US for this indication and the only treatment of any kind to be approved for GVHD in children <12 years old.

Olezarsen (Tryngolza – Ionis), an apolipoprotein C-III (apoC-III)-directed antisense oligonucleotide, has been approved by the FDA to reduce triglyceride levels in adults with familial chylomicronemia syndrome (FCS). It is the first drug to be approved in the US for this indication.

The FDA has approved a 1-mg epinephrine nasal spray (neffy - ARS Pharma) for emergency treatment of type 1 hypersensitivity reactions including anaphylaxis in patients ≥4 years old who weigh 15 to <30 kg. A 2-mg formulation of neffy was approved in 2024 for use in patients who weigh ≥30 kg.

The FDA has cleared the Lumipulse G pTau217/β-Amyloid 1-42 Plasma Ratio (Fujirebio), a bloodbased diagnostic test, for early detection of amyloid plaque pathology associated with Alzheimer's disease (AD) in adults ≥50 years old who are showing signs and symptoms of the disease. The new assay is the first blood test for amyloid biomarkers to become commercially available in the US. The Lumipulse G β-Amyloid Ratio (1-42/1-40), which measures amyloid biomarkers in cerebrospinal fluid (CSF), was cleared by the FDA in 2022.

The CD3-directed monoclonal antibody teplizumab (Tzield – Provention Bio), which was previously approved by the FDA to delay the onset of stage 3 type 1 diabetes in patients ≥8 years old with stage 2 type 1 diabetes, has now been approved for such use in children 1-7 years old.

The FDA has licensed PreHevbrio (VBI Vaccines), a recombinant, 3-antigen, 3-dose hepatitis B vaccine, for the prevention of infection caused by all known subtypes of hepatitis B virus (HBV) in adults. It has been available in Israel since 2000.

The FDA has approved tenapanor (Ibsrela - Ardelyx), a sodium/hydrogen exchanger 3 (NHE3) inhibitor, for twice-daily oral treatment of irritable bowel syndrome with constipation (IBS-C) in adults. Tenapanor is the first NHE3 inhibitor to become available in the US.

The FDA has approved tirzepatide (Mounjaro – Lilly), a peptide hormone with activity at both glucose-dependent insulinotropic polypeptide (GIP) and glucagon-like peptide-1 (GLP-1) receptors, to improve glycemic control in adults with type 2 diabetes. Tirzepatide, which is injected subcutaneously once weekly, is the first dual GIP/GLP-1 receptor agonist to become available in the US. Selective GIP receptor agonists are not available in the US; GLP-1 receptor agonists have been available for years.

The FDA has approved oteseconazole (Vivjoa – Mycovia), a new oral azole antifungal, to reduce the incidence of recurrent vulvovaginal candidiasis (RVVC) in females with a history of RVVC. Oteseconazole is the first drug to be approved in the US specifically for treatment of RVVC.

The FDA has approved the oral Janus kinase (JAK) inhibitor upadacitinib (Rinvoq – Abbvie) for treatment of moderately to severely active ulcerative colitis in adults who have had an inadequate response to or cannot tolerate one or more tumor necrosis factor (TNF) inhibitors. Upadacitinib is the second JAK inhibitor to be approved for this indication; tofacitinib (Xeljanz) was the first. Upadacitinib is also approved for treatment of rheumatoid arthritis, psoriatic arthritis, atopic dermatitis, and ankylosing spondylitis.