Šalis: Europos Sąjunga
kalba: anglų
Šaltinis: EMA (European Medicines Agency)
valoctocogene roxaparvovec
BioMarin International Limited
B02BD15
Valoctocogene roxaparvovec
Antihemorrhagics
Treatment of severe haemophilia A (congenital factor VIII deficiency) in adult patients without a history of factor VIII inhibitors and without detectable antibodies to adeno-associated virus serotype 5 (AAV5).
Revision: 3
Authorised
2022-08-24
36 B. PACKAGE LEAFLET 37 PACKAGE LEAFLET: INFORMATION FOR THE PATIENT ROCTAVIAN 2 × 10 13 VECTOR GENOMES/ML SOLUTION FOR INFUSION valoctocogene roxaparvovec This medicine is subject to additional monitoring. This will allow quick identification of new safety information. You can help by reporting any side effects you may get. See the end of section 4 for how to report side effects. READ ALL OF THIS LEAFLET CAREFULLY BEFORE YOU START USING THIS MEDICINE BECAUSE IT CONTAINS IMPORTANT INFORMATION FOR YOU. - Keep this leaflet. You may need to read it again. - If you have any further questions, ask your doctor. - If you get any side effects, talk to your doctor. This includes any possible side effects not listed in this leaflet. See section 4. - Your doctor will give you a Patient Card. Read it carefully and follow the instructions on it. WHAT IS IN THIS LEAFLET 1. What ROCTAVIAN is and what it is used for 2. What you need to know before you are given ROCTAVIAN 3. How ROCTAVIAN is given 4. Possible side effects 5. How ROCTAVIAN is stored 6. Contents of the pack and other information 1. WHAT ROCTAVIAN IS AND WHAT IT IS USED FOR WHAT ROCTAVIAN IS ROCTAVIAN is a gene therapy product that contains the active substance valoctocogene roxaparvovec. A gene therapy product works by delivering a gene into the body to correct a genetic deficiency. WHAT ROCTAVIAN IS USED FOR This medicine is used for the treatment of severe haemophilia A in adults who do not have current or past inhibitors to factor VIII and who do not have antibodies to the virus vector AAV5. Haemophilia A is a condition where people inherit an altered form of a gene needed to make factor VIII, an essential protein required for blood to clot and stop any bleeding. People with haemophilia A cannot produce factor VIII and are prone to internal or external bleeding episodes. HOW DOES ROCTAVIAN WORK The active substance in ROCTAVIAN is based on a virus which does not cause disease in humans. This virus has been modified so that it cannot spread in the body but can Perskaitykite visą dokumentą
1 ANNEX I SUMMARY OF PRODUCT CHARACTERISTICS 2 This medicinal product is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse reactions. See section 4.8 for how to report adverse reactions. 1. NAME OF THE MEDICINAL PRODUCT ROCTAVIAN 2 × 10 13 vector genomes/mL solution for infusion 2. QUALITATIVE AND QUANTITATIVE COMPOSITION 2.1 GENERAL DESCRIPTION Valoctocogene roxaparvovec is a gene therapy medicinal product that expresses the B-domain deleted SQ form of human coagulation factor VIII (hFVIII-SQ). It is a non-replicating recombinant adeno-associated virus serotype AAV5 based vector containing the cDNA of the B-domain deleted SQ form of human coagulation factor VIII gene under the control of a liver-specific promoter. Valoctocogene roxaparvovec is produced in a baculovirus expression system that derived from cells of _Spodoptera frugiperda_ (Sf9 cell line) by recombinant DNA technology. 2.2 QUALITATIVE AND QUANTITATIVE COMPOSITION Each mL of valoctocogene roxaparvovec solution for infusion contains 2 × 10 13 vector genomes. Each vial contains 16 × 10 13 vector genomes of valoctocogene roxaparvovec in 8 mL solution. Excipient with known effect This medicinal product contains 29 mg sodium per vial. For the full list of excipients, see section 6.1. 3. PHARMACEUTICAL FORM Solution for infusion. A clear, colourless to pale yellow solution with a pH of 6.9 - 7.8 and an osmolarity of 364 - 445 mOsm/L. 4. CLINICAL PARTICULARS 4.1 THERAPEUTIC INDICATIONS ROCTAVIAN is indicated for the treatment of severe haemophilia A (congenital factor VIII deficiency) in adult patients without a history of factor VIII inhibitors and without detectable antibodies to adeno-associated virus serotype 5 (AAV5). 4.2 POSOLOGY AND METHOD OF ADMINISTRATION Treatment should be initiated under the supervision of a physician experienced in the treatment of haemophilia and/or bleeding disorders. This medicinal product should be administ Perskaitykite visą dokumentą