ZOLGENSMA 20000000000000 VG — DIN 02509695

What is ZOLGENSMA?

ZOLGENSMA 20000000000000 VG contains ONASEMNOGENE ABEPARVOVEC : 20000000000000 VG. It is listed in the Health Canada Drug Product Database under DIN 02509695, held by NOVARTIS PHARMACEUTICALS CANADA INC, supplied as SOLUTION for INTRAVENOUS administration. Health Canada currently lists it as marketed, status dated 04-Feb-2021. Nexara Health is a licensed Canadian wholesale distributor (DEL 3-002896-A) and can source this product for hospitals, pharmacies and clinics. Availability and lead time are confirmed per order.

What is ZOLGENSMA used for?

Zolgensma® (onasemnogene abeparvovec) is indicated for the treatment of pediatric patients with 5q spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene and: • 3 or fewer copies of SMN2 gene; or • infantile-onset SMA. The efficacy and safety data supporting the use of Zolgensma in treating pediatric patients with SMA are derived from completed and ongoing open-label, single-arm, clinical trials in patients with: • infantile-onset SMA and 2 copies of SMN2 gene; and • presymptomatic genetically diagnosed SMA and 2 or 3 copies of SMN2 gene (see 14 Clinical Trials).

Authorized indication as written in the Health Canada product monograph. For healthcare professionals and licensed purchasers; this is not medical advice.

How is it administered?

Zolgensma should only be infused by a healthcare professional trained in the delivery of gene therapy. To improve the traceability of biological medicinal products, the name and batch number of the administered product should be clearly recorded. Zolgensma is for a single treatment only. An immune response to the adeno-associated viral vector serotype 9 (AAV9) capsid will occur after infusion of Zolgensma. Patients should not be re-dosed with Zolgensma. The recommended dose of Zolgensma is 1.1 × 10^14 vector genomes (vg)/kg patient body weight. Zolgensma is for single-dose intravenous infusion only. Preparation of Zolgensma: • Zolgensma should be prepared aseptically. • Thaw Zolgensma in the refrigerator (2°C to 8°C) for approximately 16 hours, or at room temperature (20°C to 25°C) for approximately 6 hours. • Do not use Zolgensma unless thawed. • When thawed, Zolgensma is a clear to slightly opaque, colourless to faint white liquid, free of particles. Visually inspect vials for particulate matter and discolouration prior to infusion. Do not use vials if particulates or discolouration are present. • DO NOT SHAKE. • Immediately prior to dosing, draw the prescribed dose volume from all vials into the syringe, remove air from syringe, cap syringe, and deliver filled syringe to patient infusion location. • Once dose is drawn into the syringe, it must be used within 8 hours. • DO NOT REFREEZE. Intravenous Infusion Instructions: • Place a primary catheter in a peripheral vein (generally in the arm or leg). • Insertion of a secondary catheter is recommended. • Program syringe pump for saline priming, or prime tubing manually with saline. • Administer Zolgensma as a slow infusion over 60 minutes. Do not infuse as a rapid intravenous injection or bolus. • Following completion of the infusion, flush line with saline. • Seal used Zolgensma vials in a biohazard bag and discard in biohazard waste containers according to institutional biohazard guidelines (see 11 Storage, Stability, and Disposal). Missed Dose: Not applicable. Zolgensma is to be administered only once.

Included because it drives pack selection and wastage. Consult the monograph.

Contraindications

Zolgensma is contraindicated in patients who are hypersensitive to this drug or to any ingredient in the formulation, including any non-medicinal ingredient, or component of the container. For a complete listing, see 6 Dosage Forms, Strengths, Composition, and Packaging.

Warnings and precautions

Hepatotoxicity (acute liver failure, acute liver injury and elevated liver aminotransferases) can occur with Zolgensma. Cases of acute liver failure with fatal outcomes have been reported. Patients with pre-existing liver impairment or hepatic viral infection may be at higher risk. Before infusion, assess liver function by clinical examination and by laboratory testing in all patients. Before and after infusion, administer corticosteroid (oral prednisolone or equivalent) to all patients. Do not stop systemic corticosteroids abruptly. Continue to monitor liver function for at least 3 months after infusion, and at other times as clinically indicated. Thrombotic microangiopathy (TMA) can occur with Zolgensma. Cases of TMA have been reported to occur after Zolgensma infusion in the post-market setting. Prompt attention to signs and symptoms of TMA is advised, as TMA can result in life-threatening or fatal outcomes. Before infusion, assess kidney function by laboratory testing in all patients. Monitor all patients for signs and symptoms of TMA. Patients treated with Zolgensma should not donate blood, organs, tissues or cells for transplantation. There is a theoretical risk of tumourigenicity due to integration of AAV vector DNA into the genome. The clinical relevance of individual integration events is unknown, but it is acknowledged that these events could potentially contribute to a risk of tumourigenicity. Serious infusion-related reactions, including hypersensitivity reactions and anaphylaxis, have occurred during, and/or shortly after, infusion of Zolgensma. Closely monitor patients for signs and symptoms of infusion reactions during the infusion period and immediately following the end of the infusion. If a reaction occurs, the infusion should be interrupted and treatment should be provided as needed. Based on clinical evaluation and standard practices, administration may be cautiously resumed.