dm+d

Unassigned

New Medicines

Cevenfacta (UK/EU), Sevenfact (US)Haemophilia A and B in patients with inhibitory antibodies to factor VIII or IX

Information

Cevenfacta (UK/EU), Sevenfact (US)
New molecular entity
LFB Biopharmaceuticals
HEMA Biologics

Development and Regulatory status

Approved (Licensed)
Approved (Licensed)
Launched
Oct 22Oct 22: Cevenfacta (eptacog beta[activated]) will be available in the UK on 27th October. The 2mg pack will not be available until early 2023. Pack sizes and prices will be - 1mg (45 KIU) £525.20, 2MG (90 KIU) £1050.40 and 5mg (225 KIU) £2626.00 [19].
Aug 22MHRA approves eptacog beta [18]
Jul 22Approved in EU for use in "in adults and adolescents (12 years of age and older) for the treatment of bleeding episodes and for the prevention of bleeding in those undergoing surgery or invasive procedures in the following patient groups: in patients with congenital haemophilia with high-responding inhibitors to coagulation factors VIII or IX (i.e. ≥5 Bethesda Units (BU)); in patients with congenital haemophilia with low titre inhibitors (BU <5), but expected to have a high anamnestic response to factor VIII or factor IX administration or expected to be refractory to increased dosing of FVIII or FIX [17].
May 22The company confirms eptacog beta activated is currently being reviewed by the MHRA and should be available in the UK in Q4 22. It is being considered on the NHSE Bleeding Disorder Tender so would be routinely commissioned by the NHS if accepted [16].
May 22Recommended for EU approval by CHMP for use “in adults and adolescents ≥12 years of age for the treatment of bleeding episodes and for the prevention of bleeding in those undergoing surgery or invasive procedures in the following patient groups: in patients with congenital haemophilia with high-responding inhibitors to coagulation factors VIII or IX (i.e. ≥5 Bethesda Units (BU)) and in patients with congenital haemophilia with low titre inhibitors (BU <5), but expected to have a high anamnestic response to factor VIII or factor IX administration or expected to be refractory to increased dosing of FVIII or FIX.” Cevenfacta will be available as a powder and solvent for solution for injection [15].
Apr 22Eptacog beta application is still under evaluation by the CHMP [14]
Feb 21Has been filed in the EU [10].
Apr 20FDA approves Sevenfact [coagulation factor VIIa (recombinant)-jncw] for the treatment and control of bleeding episodes occurring in adults and adolescents ≥ 12 years of age with hemophilia A or B with inhibitors (neutralizing antibodies) [9].
Jan 20Still listed as under FDA review. [8]
Jan 17The FDA has accepted the Biologic License Application (BLA) for eptacog beta activated (recombinant Factor VIIa) submitted by LFB SA, according to HEMA Biologics who have the commercialisation rights for the USA. The application is supported by the results of the PERSEPT 1 trial, the results of which were presented in abstract at the World Federation of Hemophilia July 2016 Congress [7].

Category

A transgenically produced recombinant form of human factor VIIa
Haemophilia affects 1 in 5,000 to 1 in 10,000 male live births
Haemophilia A and B in patients with inhibitory antibodies to factor VIII or IX
Parenteral

Trial or other data

Nov 21Results of PERSEPT 3 (NCT02548143) showed both eptacog beta dosing regimens were well-tolerated (n=12). The primary efficacy endpoint success proportion was 100% (95% CI: 47.8%–100%) for minor procedures and 66.7% (95% CI: 22.3%–95.7%) for major procedures; 81.8% (95% CI: 48.2%–97.7%) of the procedures were considered successful using eptacog beta [11, 13]
Aug 20Results of PERSEPT 2 (NCT02448680) demonstrated that eptacog beta treatment of bleeding episodes in the pediatric pts yielded remarkable (>95%) treatment success proportions in both initial dose regimens by 24 hours after initial eptacog beta infusion of 75 or 225 µg/kg (n=25) [11, 12]
Aug 16The PIII PERSEPT 2 trial is ongoing and still recruiting participants [5]
Aug 16The PIII PERSEPT 3 trial (NCT02548143) investigating rhFVIIa for the prevention of bleeding complications in the surgical setting in the US is ongoing, but not recruiting participants [6].
Nov 15rEVO initiates the phase III PERSEPT 2 trial in approximately 24 paediatric patients with haemophilia with inhibitors (NCT02448680). rEVO is also planning the phase III PERSEPT 3 trial to in approximately 12 male patients with congenital haemophilia A or B in the surgical setting (NCT02548143). Both the trails will be conducted in the US [4]
Aug 15The phase III PERSEPT 1 (NCT02020369) study involving 27 patients completed in August 2015. Based on an interim analysis of the safety and efficacy data rEVO plans to initiate the PERSEPT 2 and PERSEPT 3 phase III trials [3]
Mar 15Initial PIII study - Coagulation Factor VIIa, expected to complete Mar 15 [2].
Mar 14PIII programme starts. The initial PIII trial is an open-label, multicentre study of efficacy, safety and pharmacokinetics of LR769 in adolescent and adult haemophilia A and B patients with inhibitors. The study will evaluate two dosing regimens for the treatment of bleeding episodes and results are expected later this year. The second study will assess the efficacy of LR769 for the treatment of bleeding episodes in paediatric haemophilia patients with inhibitors. A third study will evaluate the prevention of bleeding complications in patients undergoing surgery. Both are expected to start early 2015 [1]