Drugs Xagena
The Committee for Medicinal Products for Human Use ( CHMP ) has adopted a positive opinion, recommending the granting of a marketing authorisation for the medicinal product Voncento.
Voncento is recommended to be used for the prevention and treatment of bleeding in patients with haemophilia A and Von Willebrand disease ( two inherited bleeding disorders ).
The indications recommended by the CHMP are as follows:
a) Von Willebrand disease ( VWD ): treatment of haemorrhage or prevention and treatment of surgical bleeding in patients with Von Willebrand disease, when Desmopressin treatment alone is ineffective or contraindicated.
b) Haemophilia A ( congenital factor VIII deficiency ): prophylaxis and treatment of bleeding in patients with haemophilia A.
The active substances in Voncento are human coagulation factor VIII and human Von Willebrand factor, substances that help the blood to clot, also known as antihaemorrhagics.
Patients with haemophilia A lack factor VIII and patients with Von Willebrand disease lack Von Willebrand factor, which leads to bleeding disorders. Voncento is used to correct the deficiency by replacing the missing coagulation factors, giving temporary control of the bleeding disorder.
Voncento is available as a powder and solvent for solution for injection or infusion in three strengths ( 250 IU / 600 IU, 500 IU / 1200 IU, and 1000 IU / 2400 IU ).
Treatment should be supervised by a doctor who has experience in the treatment of haemostatic disorders.
The Agency’s Committee for Medicinal Products for Human Use ( CHMP ) has decided that Voncento’s benefits are greater than its risks following the assessment of two main studies, one study is investigating the effects of Voncento in 81 patients with haemophilia A, and one is investigating the effects of Voncento in 22 patients with Von Willebrand disease.
Voncento has shown to be effective in preventing the occurrence of bleeding events in patients with severe haemophilia A and in stopping bleeding in patients with severe Von Willebrand disease.
When bleeding events did occur in haemophilia A patients on prophylaxis, Voncento is effective in treating these events.
Voncento was also effective in the prevention and treatment of bleeding in relation to surgery.
Regarding its safety, side effects were generally mild to moderate. The most common side effects are hypersensitivity or allergic reactions, thromboembolic events, pyrexia, headache, dysgeusia and abnormal liver function test levels.
Furthermore patients may develop inhibitors to factor VIII and Von Willebrand factor. ( Xagena )
Source: EMA, 2013
XagenaMedicine_2013
