FDA Approves Drug For Rare Blood Clotting Disorder
ByThe U.S. Food and Drug Administration announced on Monday that it has approved Novo Nordisk's drug Tretten to treat a rare blood clotting disorder, Reuters reported.
People with congenital Factor XIII A-Subunit deficiency, a genetic blood disorder, do not make enough of the Factor XIII protein that promotes normal clotting. The new drug, which is a "human recombinant" produced in yeast cells, will make up for this deficiency, HealthDay reported.
"The approval of this product provides another therapeutic option for the prevention of bleeding in patients with Factor XIII A-subunit deficiency," Karen Midthun, director of the FDA's Center for Biologics Evaluation and Research, said in a statement. "Without treatment, people with this rare condition are at risk for serious and life-threatening bleeding."
In a statement, the FDA said Tretten is a sterile freeze-dried powder "to be reconstituted with diluent and injected intravenously." The product can be administered by a physician or be self-administered.
According to Reuters the blood disorder affects about 3 million people globally and is associated with impaired wound healing and miscarriage.
The effectiveness of Tretten was studied in 77 patients with congenital Factor XIII A-subunit deficiency. During the study, researchers found that the drug was effective in preventing bleeding in 90 percent of the patients when given monthly.
Some of the side effects reported in this study were headache, pain in the extremities and pain at injection site. No individual in the trial developed abnormal clots.
The drug is expected to generate sales of $84 million by 2018, according to the average estimate of three analysts polled by Thomson Reuters.
The product was approved last year in Europe and Canada.