FDA approves novel treatment targeting abnormality in sickle cell disease
The FDA approved voxelotor (Oxbryta) for the treatment of sickle cell disease (SCD) in adults and pediatric patients 12 years of age and older. Voxelotor, an inhibitor of deoxygenated sickle hemoglobin polymerization, has been shown to inhibit red blood cell sickling, improve the ability of red blood cells to change shape, and improve the blood's ability to flow. With voxelotor, sickle cells are less likely to bind together and form the sickle shape, which can cause low hemoglobin levels due to red blood cell destruction.
Voxelotor was approved based on results of a clinical trial of 274 SCD patients randomly assigned to voxelotor or placebo. Treatment with voxelotor led to clinically meaningful and statistically significant improvements in A1C levels, accompanied by reductions in hemolysis. After 24 weeks of treatment, 51.1% of patients receiving voxelotor achieved greater than 1 g/dL increase in hemoglobin compared with 6.5% in the placebo group (P < .001).
Approval for voxelotor was granted to Global Blood Therapeutics.
Treatment approved for partial-onset seizures in adults with epilepsy
The FDA approved cenobamate (Xcopri) for the treatment of partial-onset seizures in adults with epilepsy. The approval was based on results from two randomized controlled trials that included over 600 patients; the trial showed that cenobamate significantly reduced the percentage of seizures compared with placebo at doses of 100 mg, 200 mg, and 400 mg. Although the recommended maintenance dose of cenobamate is 200 mg/day after titration, some patients may need to be titrated up to 400 mg/day.
The most common adverse reactions in the trials included somnolence, dizziness, fatigue, and diplopia. Most treatment-emergent adverse reactions were reported to be mild-to-moderate in severity; however, one participant in a study who received 200 mg cenobamate experienced drug reaction with eosinophilia and systemic symptoms (or multiorgan hypersensitivity). Another patient in a different trial died when the active drug was titrated rapidly. A higher percentage of patients who took cenobamate had a shortening of the QT interval of greater than 20 milliseconds compared with placebo. Consult product labeling for details.
Cenobamate is a product of SK Life Sciences.
New antibacterial approved to treat cUTIs
Cefiderocol (Fetroja) was FDA-approved to treat complicated urinary tract infections (cUTIs), including kidney infections caused by susceptible Gram-negative microorganisms in patients 18 years of age or older who have limited or no alternative treatment options.
The safety and efficacy of cefiderocol were shown in a study of 448 patients with cUTIs. Of patients given the drug, 72.6% had resolution of symptoms and eradication of the bacteria approximately 7 days after completing treatment, compared with 54.6% in patients who received an alternative antibiotic. Clinical response rates were similar between the two treatment groups.
Cefiderocol carries a warning label regarding the higher all-cause mortality observed in cefiderocol-treated patients compared with those treated with other antibiotics in a trial with critically ill patients who had multidrug-resistant Gram-negative bacterial infections. The cause for this has not been established.
Cefiderocol is a product of Shionogi. Consult product labeling for detailed prescribing information.