Novo Nordisk has won US approval for Tretten, the first recombinant treatment for a rare clotting disorder known as congenital Factor XIII A-subunit deficiency.
Tretten (catridecacog) has been cleared by the FDA, which has granted it orphan drug designation, for the routine prophylaxis of bleeding in adults and children with the disorder, which affects one in around 2-5 million people worldwide.
Novo had received a complete response letter (CRL) from the FDA asking for more data on manufacturing in August, but resubmitted its application shortly thereafter.
People with the deficiency are generally diagnosed in infancy and suffer serious bleeds, with a high risk of intracranial haemorrhage that can be life-threatening. Currently, injections with plasma-derived Factor XIII concentrates every 4 to 6 weeks are used to try to prevent spontaneous bleeding.
Karen Midthun, director of the FDA’s Center for Biologics Evaluation and Research, said the approval “provides another therapeutic option for the prevention of bleeding in patients with Factor XIII A-subunit deficiency.”
The effectiveness of Novo’s therapy was evaluated in 77 patients with the disorder. Study findings demonstrated that monthly treatment with Tretten prevented bleeding in 90 per cent of patients, with no evidence of abnormal clotting.
Novo Nordisk said Tretten is expected to be launched in the US early next year. The product is already sold in Canada under the same brand name, and in the EU, Switzerland and Australia as NovoThirteen.
The company indicated that it has filed the recombinant analogue for approval in a number of other countries. Given the patient population for Tretten/NovoThirteen is so tiny sales are expected to be modest, although analysts at Jyske Bank predicted recently it could eventually achieve revenues of around $90m a year.
Tretten/NovoThirteen is one of a series of new drugs Novo is developing to reinvigorate its clotting disorder franchise as its established products lose patent protection and the sector gets increasingly competitive, with new entrants from the likes of Biogen Idec.
In October, Novo secured US approval for NovoEight, a new long-acting Factor VIII for people with haemophilia A (turoctocog alfa), while it also has NN7999, a long-acting recombinant coagulation Factor IX derivative for haemophilia B, in phase III development.
The company suffered a disappointment last year when it was forced to drop a long-acting Factor VIIa therapy called vatreptacog alfa after patients in trials developed inhibitory antibodies to the drug.
