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Safety in Lactation: Drugs for bleeding disorders

18 September 2020Additional information relating to breastfeeding To be used in conjunction with individual drug entries for specific information and guidance. Antifibrinolytics & haemostatics Etamsylate is a…
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New Medicines

EsperoctHaemophilia A - treatment and prophylaxis of bleeding in patients aged ≥12 years

Information

Esperoct
New formulation
Novo Nordisk
Novo Nordisk

Development and Regulatory status

Launched
Launched
Launched
July 2020
Yes
Jul 20Available in the UK [15].
Jun 20Will be available from July 2020. Pricing 1 vial x 500IU = £425, 1 vial x 1000IU = £850, 1 vial x 1500IU = £1275, 1 vial x 2000UI = £1700, 1 vial x 3000IU = £2550 [15].
Feb 20Novo Nordisk launches Esperoct in the US for the treatment of people with hemophilia A [13].
Jun 19Approved in EU [12].
Apr 19The CHMP has adopted a positive opinion for the use of Esperoct® (turoctocog alfa pegol, N8-GP) recommending marketing authorisation for the treatment of adolescents and adults with haemophilia A [10].
Apr 19The full indication recommended by CHMP is “treatment and prophylaxis of bleeding in patients 12 years and above with haemophilia A (congenital factor VIII deficiency)”. It is proposed that the product be prescribed by physicians experienced in the treatment of haemophilia [11].
Feb 19FDA approves treatment for haemophilia A. Expected to be available in US in 2020 [9].
Feb 18Filed in EU and US [8].
Feb 17Novo Nordisk intends to file in the EU and US in 2018 [7].
Jan 17PIII development continues [6].
Oct 15PIII development continues [5].
Jun 12Orphan drug status granted in the EU (EU/3/12/995) [2].
Feb 12PIII 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 - treatment and prophylaxis of bleeding in patients aged ≥12 years
Intravenous

Trial or other data

Mar 14PathfinderT2 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 13Open-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 12PIII (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 11NCT01480180 (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].

Evidence based evaluations