The results of a clinical trial (PRECISION) comparing the cardiovascular safety of the COX-2 selective NSAID celecoxib (Celebrex, and generics) with that of ibuprofen and naproxen, which are nonselective, have been described in the lay press in terms that may overestimate the safety of celecoxib.

Exagamglogene autotemcel (Casgevy – Vertex), a cell-based gene therapy recently approved for treatment of sickle cell disease1, has now been approved by the FDA for treatment of patients ≥12 years old with transfusion-dependent beta thalassemia. Casgevy is the first gene therapy that uses CRISPR/Cas9 gene-editing technology to be approved in the US for any disorder. Betibeglogene autotemcel (Zynteglo), an autologous lentiviral vector cell-based gene therapy, was approved in the US in 2022 for treatment of transfusion-dependent beta thalassemia.

Palivizumab (Synagis - MedImmune), an injectable monoclonal antibody, has been marketed in the USA since 1999 for prevention of respiratory syncytial virus (RSV) disease in high-risk infants and children.

The heptavalent pneumococcal conjugate vaccine (Prevnar - Wyeth) previously approved by the FDA for prevention of invasive pneumococcal disease in infants and children (Medical Letter 2000; 42:25) has now also been approved for prevention of otitis media. Infants and young children have higher antibody responses to the heptavalent conjugate vaccine (PCV7) than to the older 23-valent pneumococcal polysaccharide vaccine (PPV23) used in adults and older children (D Murray and C Jackson, Mil Med 2002; 167:671).

Agalsidase beta (Fabrazyme Genzyme) has received accelerated approval from the FDA for treatment of patients with Fabry disease, an inherited lysosomal storage disease caused by deficiency of α-galactosidase A. Agalsidase beta is a recombinant form of human α-galactosidase A. This review provides an overview of this rare disease, including its clinical manifestations. The clinical trials conducted with the new drug are also described, as well as its adverse effects, dosage, and cost. Appropriate indications for use of Fabrazyme are summarized.

The FDA has approved Humatrope, Lilly's brand of recombinant human growth hormone (somatropin), for long-term treatment of children with idiopathic, non-growth-hormone-deficient (NGHD) short stature who are more than 2.25 standard deviations below the mean height for their age and sex. This review describes the clinical studies, adverse effects, and includes a cost table for other brands of somatropin.

Solar ultraviolet (UV) light capable of injuring the skin is classified by wavelength into UVA I (340-400 nm), UVA II (320-340 nm) and UVB (290-320 nm). UVB is responsible for most of the erythema of sunburn. UVA has been implicated in the development of phototoxicity and photoaging. The FDA permits sunscreen manufacturers to claim broad-spectrum protection if their products block at least part of UVA II in addition to UVB.

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The ViewPoint CK System (Refractec) for conductive keratoplasty (CK), previously approved by the FDA as a device for treatment of hyperopia (farsightedness), was recently approved for treatment of presbyopia (age-associated loss of ability to view near objects).

The FDA has approved Eloctate (Biogen), a recombinant factor VIII Fc fusion protein with an extended half-life, for use in adults and children with hemophilia A. Eloctate is indicated for control and prevention of bleeding episodes, perioperative management, and routine prophylaxis to prevent or reduce the frequency of bleeding episodes. It is the first antihemophilic factor VIII Fc fusion protein to be approved for hemophilia A.

The FDA has approved donislecel-jujn (Lantidra – CellTrans), an allogeneic pancreatic islet cellular therapy, for use in conjunction with immunosuppression for treatment of adults with type 1 diabetes who are unable to approach target HbA1c because of current repeated episodes of severe hypoglycemia despite intensive diabetes management and education. Donislecel is the first cell-based treatment to be approved in the US for type 1 diabetes.