Turoctocog alfa pegol

Unassigned

New Medicines

Haemophilia A - prophylaxis and treatment of bleeds

Information

New formulation
Novo Nordisk
Novo Nordisk

Development and Regulatory status

Pre-registration (Filed)
Pre-registration (Filed)
Pre-registration (Filed)
Yes

Feb 18. Filed in EU and US [8]


Feb 17: Novo Nordisk intends to file in the EU and US in 2018 [7]


Jan 17. PIII development continues [6].


Oct 15: PIII development continues [5].


Jun 12: Orphan drug status granted in the EU (EU/3/12/995) [2]


Feb 12 - PIII study to start [1]

Category

A long acting recombinant factor VIII derivative. NN 7088 is a glycopegylated protein based on GlycoPEGylation™ technology.
0.7 in 10,000 people in the EU have haemophilia A - equivalent to around 35,000 people [2].
Haemophilia A - prophylaxis and treatment of bleeds
Intravenous

Trial or other data

Mar 14: PathfinderT2 has been completed. In the trial, 175 patients were treated with a prophylactic regimen of 50U/kg every 4th day and 11 patients had on-demand treatment when bleedings occurred. Patients were treated for up to 21 months, resulting in median annualised bleeding rates of 1.3 and 30.9 episodes for patients treated prophylactically and on-demand, respectively. Pharmacokinetic data documented a single dose half-life of 18.4 hours and a mean trough level of 8% measured immediately before next dose for patients on prophylaxis treatment. N8-GP appeared to have a safe profile and to be well tolerated. Among the 186 patients in the trial, one patient who responded well to prophylactic treatment throughout the trial developed an FVIII inhibitor. Novo Nordisk is expecting the three remaining trials in the pathfinderT programme to be finalised within the next 12 months [4]


Feb 13: Open-label, single-arm PIII (NCT01731600) pathfinder 5 study to investigate safety, efficacy & pharmacokinetics of NNC 0129-0000-1003 (N8-GP) in children aged up to 11 years with severe haemophilia A who have undergone treatment with previous factor VIII (FVIII) products begins. 60 children will be recruited from the US, Brazil, Germany, Italy, Switzerland & Ukraine. Pts will receive a fixed dose of N8-GP iv twice weekly for prophylaxis. In addition, N8-GP will be administered to treat bleeding episodes during the trial period. Bleeding episodes will be treated with doses of 20-75 U/kg body weight. The primary outcome measure is incidence of inhibitory antibodies against coagulation factor VIII (FVIII) equal to or above 0.6 Bethesda units. The study is expected to complete collection of data for the primary outcome in Aug 14 [3].


Aug 12: PIII (NCT01489111) pathfinder 3 study began recruiting 18 pts from sites worldwide (including US, EU & UK) to evaluate the haemostatic effect of NNC 0129-0000-1003 during surgical procedures in subjects with haemophilia A. Inclusion crtieria include age 12 & over, male, ongoing participation in the pathfinder™2 (NN7088-3859) or the pathfinderTM 4 (NN7088-3861) trials, & having received greater than or equal to 5 doses of N8-GP, & undergoing major surgery requiring daily monitoring of FVIII:C (FVIII activity) & wound status for at least 3 days. The primary outcome is haemostatic effect during surgery evaluated by the four-point scale (excellent, good, moderate or none). The study is expecetd to complete in Sep 13 [3].


Nov 11: NCT01480180 (pathfinder 2) is a PIII open-label multi-national trial evaluating safety efficacy and pharmacokinetics of NNC 0129-0000-1003 for treatment and prophylaxis of bleeding in 132 patients with haemophilia A. The primary outcomes are: incidence rate of FVIII-inhibitors ≥ 0.6 BU at study end (~19 months) and annualised bleeding rate in the prophylaxis arm. The study will start Feb 12 and is due complete Oct 13. Inclusion criteria include males, aged 12 or over, with severe congenital haemophilia A (FVIII activity <1%) and a documented history of ≥150 exposure days to other FVIII products [1].