Orphan Drugs: FDA Approval Of Tretten For Rare Genetic Clotting Disorder
On December 23, the FDA announces the approval of Novo Nordisk’s orphan drug Tretten (Coagulation Factor XIII A-Subunit Recombinant), for the prevention of bleeding in adults and children who have the rare genetic blood clotting disease, Congenital Factor XIII A-subunit deficiency. Tretten is a sterile freeze-dried powder “to be reconstituted with diluent and injected intravenously.”
Patients with this rare clotting disorder, do not make enough Factor XIII, a blood component that is important for blood clotting. The National Hemophilia Foundation estimates the incidence at 1/5,000,000 births. According to 2011 estimates, only 1,054 patients are diagnosed worldwide, of which an estimated 108 live in the United States. Tretten, a human recombinant produced in yeast cells, makes up for this deficiency. The FDA approval of Tretten is based on a Phase III clinical trial that shows “that when compared to an historic control group of individuals who did not receive routine FXIII infusions, preventive treatment with monthly 35 IU/kg TRETTEN injections significantly decreased the number of treatment-requiring bleeding episodes”.
Tretten is expected to be available in the United States in early 2014. Tretten is also :
• Approved in Canada as Tretten
• Approved in the EU, Switzerland, & Australia as NovoThirteen.
Please Note: FDA Official Logo from FDA website.