LFB announces the approval of CEVENFACTA® (eptacog beta) in the European Union

The first new bypassing agent approved in the European Union for the treatment and control of bleeding episodes in haemophilia A and B patients with inhibitors in over 2 decades.
This marketing authorization in the European Union follows the FDA approval in April 2020 of SEVENFACT® (another brand name of eptacog beta) in the United States for the treatment of bleeding episodes in adults and adolescents with haemophilia A or B with inhibitors.
In addition to this marketing authorization in the United States and in the European Union, LFB has been granted a Marketing Authorization for SEVENFACT® in Mexico on June 2nd, 2022.
The marketing authorization file has also been submitted to the UK authorities (MHRA) and the file is currently under review.

LFB today announced that the European Medicines Agency (EMA) has granted on July 15th a Marketing Authorisation for CEVENFACTA® (eptacog beta), as the first new bypassing agent in over 20 years. CEVENFACTA® is indicated 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.

About PERSEPT studies
* The approval of CEVENFACTA® was based on data from the phase III clinical trials, PERSEPT 1 and PERSEPT 3. The PERSEPT 1 Phase III, multicentre, randomised, open-label crossover study of two initial dose regimens (75µg/kg and 225µg/kg), evaluated 468 bleeding episodes across the full type of severity of bleeding episodes (mild, moderate, and severe), in 27 adolescent and adult haemophilia A and B patients with inhibitors (12-54 years of age). Both dosing regimens met the primary endpoint with 81% and 90% of bleeds controlled at 12 hours with the 75µg/kg dose and the 225µg/kg dose respectively. By 24 hours, haemostatic efficacy (secondary endpoint) was retained in 96.7% of bleeding episodes treated with the 75 µg/kg dose regimen and 99.5% of redundancy bleeding episodes treated with the 225 µg/kg dose, without requiring any alternative therapy. The median time to attain haemostatic efficacy was 5.98 hours for the 75 µg/kg dosing regimen and 3 hours for the 225 µg/kg dosing regimen. A median of 2 injections was needed to treat a bleeding episode with the 75 µg/kg and a median of only 1 injection of the 225 µg/kg dosing regimen was needed. The PERSEPT 3 Phase III, multicentre, open-label, single-arm study evaluated the safety and efficacy of CEVENFACTA® in haemophilia A or B patients with inhibitors who were scheduled for an elective surgical or other invasive procedure. 12 patients were enrolled in the study, 6 with minor procedures and 6 with major procedures. For major surgical/invasive procedures, treatment was administered at an initial bolus dose of 200 µg/kg immediately before the start of the invasive procedure. For a minor elective surgical procedure, an initial bolus dose of 75 µg/kg was administered immediately before the start of the procedure. Overall, 81.8% of procedures were reported as successfully treated at 48 hours after the last administration of the product. No thromboembolic events were reported in these two clinical trials. No Serious Adverse Events (SAEs) were considered as related to the treatment. Patients should be monitored for any signs of thrombosis, hypersensitivity and neutralising antibodies. The most frequently reported adverse reactions in studies were infusion site discomfort, infusion site haematoma, post-procedural haematoma, infusion related reaction, increased body temperature, dizziness and headache.