Etuvetidigene autotemcel

WASKYRA ® is indicated for the treatment of pediatric patients aged 6 months and older and adults with Wiskott-Aldrich Syndrome (WAS) who have a mutation in the WAS gene and for whom hematopoietic stem cell transplantation (HSCT) is appropriate and no suitable human leukocyte antigen (HLA)-matched related stem cell donor is available. WASKYRA ® is an autologous hematopoietic stem cell-based gene therapy indicated for the treatment of pediatric patients aged 6 months and older and adults with Wiskott-Aldrich Syndrome (WAS) who have a mutation in the WAS gene for whom hematopoietic stem cell transplantation (HSCT) is appropriate and no suitable human leukocyte antigen (HLA)-matched hematopoietic related stem cell donor is available.

WASKYRA ® adds full-length copies of the human WAS complementary deoxyribonucleic acid (cDNA) into patients’ hematopoietic stem cells (HSCs) through transduction with WAS lentiviral vector (LVV). After infusion, the genetically modified cells engraft in the bone marrow, repopulate the hematopoietic compartment, and produce biologically active lymphoid and myeloid progenitors whose progeny express WAS protein (WASP). WASP regulates the structural protein actin in blood cells.

For autologous use only. For single-dose intravenous use only. For intravenous use only. Patients are required to undergo hematopoietic stem and progenitor cell (HSPC) mobilization followed by apheresis to obtain CD34 + cells for WASKYRA ® manufacturing. Dosing of WASKYRA ® is based on the number of CD34 + cells in the infusion bag(s) per kg of body weight at the time of infusion. The minimum recommended dose is 7 x 10 6 CD34 + cells per kg. Reduced-intensity conditioning is required before infusion of WASKYRA ®. Prior to WASKYRA ® infusion, confirm that the patient’s identity matches the essential unique patient information on the infusion bag(s).

Hypersensitivity to the active substance or to any of the excipients. Previous treatment with HSCT within 6 months prior to treatment or HSCT with evidence of residual donor cells. Previous treatment with hematopoietic stem cell gene therapy. Contraindications to the mobilization and the conditioning regimen. Previous treatment with HSCT within 6 months prior to screening or HSCT with evidence of residual donor cell.

Hypersensitivity and infusion-related reactions: Monitor patients for hypersensitivity and infusion-related reactions during and after infusion. Engraftment failure: Monitor patients for signs and symptoms of engraftment failure. In case of engraftment failure, infuse the non-transduced back-up hematopoietic stem cells according to local standards. Cytopenias: Severe cytopenias, including anemia, neutropenia, and thrombocytopenia have occurred for several weeks following reduced intensity conditioning and WASKYRA ® infusion. Monitor patients for signs and symptoms of cytopenia for at least 8 weeks after treatment with WASKYRA ® and manage accordingly. Serious infections: Serious infections have occurred with WASKYRA ® administration. Increased susceptibility to infections may occur due to concomitant administration of rituximab and conditioning regimen.

The most common adverse reactions (incidence ≥ 20%) are catheter related infections, bacterial and viral infections, diarrhea, vomiting, stomatitis, liver injury, head injury, rhinitis, cough, rash, petechiae, hypersensitivity, anemia, febril neutropenia, epistaxis, pyrexia, catheter site complications.

No formal drug interaction studies have been performed. WASKYRA ® is not expected to interact with the hepatic cytochrome P-450 family of enzymes or drug transporters.

Store WASKYRA ® in the vapor phase of liquid nitrogen at less than -130°C (-202°F) until ready for thaw and administration. Do not re-freeze after thawing.