Source: FDA, National Drug Code (US) Revision Year: 2025
WASKYRA (etuvetidigene autotemcel) is an autologous hematopoietic stem cell-based gene therapy for intravenous infusion.
WASKYRA is prepared from the patient's own hematopoetic stem cells (HSCs), which are collected using apheresis procedure(s). The autologous cells are enriched for CD34+ cell and then transduced ex vivo with a replication incompetent self-inactivating (SIN) human immunodeficiency virus-1 (HIV-1)-based lentiviral vector (LVV) that has been modified to carry the WAS gene sequence under the control of the human WAS promoter. The transduced CD34+ cells are washed, formulated into a suspension, and then cryopreserved.
WASKYRA is manufactured for each individual patient into infusion bags, which are cryopreserved before being thawed prior to administration [see Dosage and Administration (2.3), How Supplied/Storage and Handling (16)].
The formulation contains 7% (w/v) human serum albumin (HSA) and 5% (v/v) dimethyl sulfoxide (DMSO). Each 1mL of WASKYRA suspension for IV infusion contains 3.5 mg of sodium.
| Dosage Forms and Strengths |
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WASKYRA is a single-dose cell suspension for intravenous infusion. WASKYRA is provided in one to eight infusion bags which contain 2–11.4 x 106 cells/mL (1.9–11.4 x 106 CD34+ cells/mL) [see How Supplied/Storage and Handling (16)]. Each infusion bag contains 10 to 20 mL of WASKYRA. The thawed product is colorless to yellow or pink and may be cloudy to clear. See the Lot Information Sheet for actual dose. |
| How Supplied |
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WASKYRA is supplied in 1 to 8 infusion bags containing a frozen suspension of genetically modified autologous cells enriched for CD34+ cells. Each bag contains 10 to 20 mL of suspension. Infusion bags consist of a 50 mL ethylene vinyl acetate (EVA) bag(s) with two available spike ports, packed in an EVA overwrap bag placed inside a metal cassette. WASKYRA is shipped from the manufacturing facility to the treatment centre storage facility in a cryoshipper, which may contain multiple metal cassettes intended for a single patient. Each metal cassette contains one infusion bag with WASKYRA. A Lot Information Sheet and the Chain of Custody/Chain of Identity is included with the cryoshipper. 50 mL infusion bag, overwrap, and metal cassette (NDC XXXXX-XXXX-X). Match the identity of the patient with the patient identifiers, and Lot Information Sheet upon receipt. Manufactured for: Fondazione Telethon ETS, Via Varese 16/B, 00185 Rome, Italy |
| Drug | Countries | |
|---|---|---|
| WASKYRA | Lithuania, United States |
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