IntravenousSpinal muscular atrophyChild: For patients with bi-allelic mutation in the survival motor neuron 1 (SMN1) gene and with either a clinical diagnosis of spinal muscular atrophy (SMA) Type 1 or patients with up to 3 copies of the survival motor neuron 2 (SMN2) gene: <2 years 1.1 x 1014 vector genomes/kg as a single dose via slow IV infusion over approx 60 minutes. Refer to specific product guidelines for further information on the recommended dosing values based on weight. Initiate an immunomodulatory regimen 24 hours before the infusion, then continue for ≥30 days to help prevent hepatic toxicity (refer to specific product guidelines for further information on the concomitant therapy).
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Patient with acute hepatic viral illness, concurrent infection. Postpone treatment in patients with active infections until clinically stable or infection has resolved. Hepatic impairment. Infants <2 years.
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Significant: Hepatotoxicity manifested by elevated transaminases (e.g. ALT and AST levels); increased cardiac troponin-I levels; increased risk of serious systemic immune response; antibody development.
Gastrointestinal disorders: Vomiting.
General disorders and administration site conditions: Fever.
Potentially Fatal: Acute liver failure and acute serious liver injury; thrombocytopenia, thrombotic microangiopathy, acute kidney injury, microangiopathic haemolytic anaemia.
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Baseline: Monitor liver function (e.g. clinical exam, AST, ALT, total bilirubin, and prothrombin time), CBC (e.g. platelet, Hb), serum creatinine, troponin-I; conduct an anti-adeno-associated virus serotype (AAV9) antibody test. 1st month after treatment: Monitor liver function (same as baseline), platelets; and troponin-I (weekly). Assess for signs and symptoms of thrombotic microangiopathy. 2nd-3rd month after treatment: Monitor liver function (same as baseline) every other week until AST/ALT are <2 times the ULN and clinical exam, prothrombin time, and total bilirubin are normal; platelet count every other week until counts return to baseline; troponin-I monthly until level returns to baseline. Continue to assess for signs and symptoms of thrombotic microangiopathy.
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Description:
Mechanism of Action: Onasemnogene abeparvovec, a non-replicating recombinant adeno-associated virus serotype (AAV9) vector, is a gene therapy designed to deliver a functional copy of the gene that encodes the human survival motor neuron (SMN) protein. It provides an alternative source of SMN protein expression, which is expected to stimulate the function and survival of the transduced motor neurons.
Pharmacokinetics:
Distribution: Vector DNA levels are detected in the heart, brain, inguinal lymph node, lung, intestines, kidney, pancreas, peripheral nerves, skeletal muscles, spinal cord, spleen, thymus, and with highest concentrations in the liver.
Excretion: Mainly via faeces; urine and saliva.
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Transport and/or delivery: Store vials at ≤-60°C. Upon receipt: Store vials between 2-8°C for ≤14 days. Do not refreeze.
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M09AX09 - onasemnogene abeparvovec ; Belongs to the class of other drugs for disorders of the musculo-skeletal system.
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Anon. Onasemnogene Abeparvovec. AHFS Clinical Drug Information [online]. Bethesda, MD. American Society of Health-System Pharmacists, Inc. https://www.ahfscdi.com. Accessed 10/09/2024. Brayfield A, Cadart C (eds). Onasemnogene Abeparvovec. Martindale: The Complete Drug Reference [online]. London. Pharmaceutical Press. https://www.medicinescomplete.com. Accessed 10/09/2024. Novartis New Zealand Limited. Zolgensma Injection for Intravenous Infusion data sheet 7 February 2024. Medsafe. http://www.medsafe.govt.nz. Accessed 10/09/2024. Onasemnogene Abeparvovec. UpToDate Lexidrug, Lexi-Drugs Multinational Online. Waltham, MA. UpToDate, Inc. https://online.lexi.com. Accessed 10/09/2024. Zolgensma (Novartis Gene Therapies, Inc.). DailyMed. Source: U.S. National Library of Medicine. https://dailymed.nlm.nih.gov/dailymed. Accessed 10/09/2024. Zolgensma Solution for Infusion (Novartis Pharmaceuticals [HK] Ltd). MIMS Hong Kong. http://www.mims.com/hongkong. Accessed 10/09/2024. Zolgensma Solution for Infusion (Novartis Pharmaceuticals UK Limited). MHRA. https://products.mhra.gov.uk. Accessed 10/09/2024. Zolgensma Suspension for Intravenous Infusion (Novartis Corporation [Malaysia] Sdn. Bhd.). National Pharmaceutical Regulatory Agency - Ministry of Health Malaysia. https://www.npra.gov.my. Accessed 10/09/2024.
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