Date: 2018-10-18
Type of information: Positive opinion for the granting of a Market Authorisation in the EU
Product name: NovoSeven® RT (eptacog alfa)
Compound: coagulation factor VIIa
Therapeutic area: Hematologic diseases - Genetic diseases - Rare diseases
Action mechanism:
Company: Novo Nordisk (Denmark)
Disease:
- prevention and treatment of bleeding episodes in those
undergoing surgery or invasive procedures in the following patient groups:
- -in patients with congenital haemophilia with inhibitors to coagulation factors VIII or IX > 5 Bethesda Units (BU)
- -in patients with congenital haemophilia who are expected to have a high anamnestic response to factor VIII or factor IX administration
- -in patients with acquired haemophilia
- -in patients with congenital FVII deficiency
- -in patients with Glanzmann’s thrombasthenia with past or
present refractoriness to platelet transfusions, or where platelets are not readily available
Latest news:
- • On October 18, 2018, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive
opinion recommending a change to the terms of the marketing authorisation for NovoSeven®. The CHMP adopted an extension to an existing indication as follows:
“NovoSeven® is indicated 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 inhibitors to coagulation factors VIII or IX > 5 Bethesda Units (BU)
- -in patients with congenital haemophilia who are expected to have a high anamnestic response to factor VIII or factor IX administration
- -in patients with acquired haemophilia
- -in patients with congenital FVII deficiency
- -in patients with Glanzmann’s thrombasthenia with past or
present refractoriness to platelet transfusions, or where platelets are not readily available.”
- • On July 7, 2014, Novo Nordisk announced the FDA has approved NovoSeven® RT (Coagulation Factor VIIa [Recombinant]) as the first recombinant treatment for bleeding episodes and perioperative management in patients with Glanzmann's Thrombasthenia (GT) with refractoriness to platelet transfusions, with or without antibodies to platelets. The safety and effectiveness of NovoSeven® RT were demonstrated in the treatment of severe bleeding episodes and the perioperative management for major and minor surgical procedures for this specific patient population. Patients with GT have a lifelong susceptibility toward bleeding episodes, including frequent, severe, and potentially life-threatening hemorrhages. The condition, which affects one in 1 million people globally, occurs because certain surface proteins on platelets are missing or do not work, significantly impacting the blood\'s ability to form strong clots Patients with GT may be administered platelet transfusions when experiencing severe bleeding or when surgical procedures are required. However, some patients do not respond well or at all to platelet transfusions (called refractoriness). The FDA approved NovoSeven® RT for the treatment of GT based upon evidence collected from the global Glanzmann\'s Thrombasthenia Registry (GTR) (218 patients with 1,073 bleeding and surgical events) and the Hemostasis & Thrombosis Research Society Registry (7 patients with 23 bleeding episodes). The data supported the effectiveness and safety of NovoSeven® RT (90 mcg/kg) in the treatment of bleeding and for the prevention of bleeding during surgical procedures in patients with GT.
- Overall, the GTR collected data from 92 patients treated with NovoSeven® RT for 266 severe bleeding episodes and 77 patients treated with NovoSeven® RT for 160 surgical and other invasive procedures. Treatment with NovoSeven® RT was successful in 94.4 percent of bleeding episodes and 99.4 percent of surgical procedures, based upon review of data by independent hematology experts. Of 140 patients treated for 518 bleeding episodes, surgeries, or traumatic injuries, the following adverse reactions were reported: deep vein thrombosis (n=1), headache (n=2), fever (n=2), nausea (n=1), and dyspnea (n=1).
Patents:
Submission of marketing authorization application USA :
Submission of marketing authorization application UE:
Withdrawal of marketing authorization application USA:
Withdrawal of marketing authorization application UE:
US authorization: 2014-07-07
UE authorization: 1996-02-23 (first approval) - 2006-02-23 (latest renewal)
Favourable opinion UE:
Favourable opinion USA:
Orphan status USA: 2004-06-18
Orphan status UE:
Pediatric exclusivit _USA:
Pediatric exclusivity UE:
OTC status:
Other news:
Is general: Yes
