Catridecacog

Unassigned

New Medicines

NovoThirteen (EU), Tretten (US) · Congenital factor XIII A-subunit deficiency

Information

NovoThirteen (EU), Tretten (US)
New molecular entity
Novo Nordisk
Novo Nordisk

Development and Regulatory status

Licensed but not launched
Launched
Launched
Jan 17 · Novo Nordisk has no current plans to launch in the UK [13].
Mar 14 · Has been launched for Factor XIII deficiency in the US [12].
Mar 14 · Licence extension for use in children under the age of 6 years approved in the EU [10].
Jan 14 · EU positive opinion for a licence extension to include long-term prophylactic treatment of bleeding in adult and paediatric patients with congenital factor XIII A-subunit deficiency [9].
Dec 13 · Approved in the US for routine prophylaxis of bleeding in adults and children with congenital Factor XIII A-subunit deficiency [7]
Aug 13 · The FDA has issued a second complete response letter citing unresolved findings at the facility where the company manufactures the drug [6].
Mar 13 · Novo Nordisk expected to launch catridecacog in the first European countries towards the end of 2012 [5].
Feb 13 · Launched in Denmark [8].
Sep 12 · Approved in the EU [4].
May 12 · EU positive opinion for long-term prophylactic treatment of bleeding in patients 6 years and above with factor XIII A-subnunit deficiency [1].
Feb 11 · Filed in the US [2].

Category

A recombinant coagulation factor XIII A-subunit. It is structurally identical to the human FXIII A-subunit [A2], the subunit of FXIII which contains the enzymatic activity, which binds to the FXIII B-subunit [A2B2].
Congenital factor XIII deficiency is an inherited bleeding disorder due to reduced levels & activity of factor XIII (FXIII) & characterised by hemorrhagic diathesis frequently associated with spontaneous abortions & defective wound healing. Factor XIII deficiency is one of the most rare coagulation factor deficiencies. Prevalence of homozygous forms is estimated at around 1 per 2,000,000. Men & women are affected equally [3].
Congenital factor XIII A-subunit deficiency
Intravenous

Trial or other data

Dec 10 · Positive results from a PIII trial in 41 pts in the US, Canada & Europe presented (NCT00713648). When compared with a historic control gp of individuals who did not receive recombinant factor XIII infusions, preventive treatment with monthly catridecacog injections significantly decreased the number of bleeding episodes requiring treatment with an additional factor XIII product (primary endpoint). Pts received intravenously administered catridecacog once every four weeks at a dose of 35 IU/kg. Over the study period, a total of five bleeding episodes, all associated with trauma and requiring additional FXIII treatment, were observed in four patients. No thromboembolic events or fatal adverse events were reported. An extension of this trial (NCT00978380) is ongoing and will continue until the product becomes available in the market. However, an interim analysis will be conducted when all subjects have completed 52 weeks in the trial [2].

Evidence based evaluations