Your purchase entitles you to full access to the information contained in our drug profile at the time of purchase. This information will allow us to better understand how AdisInsight is being used. How to enable JavaScript in your browser? Mechanism of Action Gene transference; Survival of motor neuron 1 protein replacements Orphan Drug Status Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases. endstream
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Indicated for gene replacement therapy in children aged <2 years with spinal muscular atrophy (SMA) type 1 (also called Werdnig-Hoffman disease) who have biallelic mutation in the survival motor neuron 1 (SNM1) gene, ≥2 years: Safety and efficacy not established, Administered as a one-time, single IV infusion through a venous catheter, 1.1 x 10^14 vector genomes per kilogram (vg/kg) of body weight, Elevated aminotransferases (>ULN) (27.3%), Acute serious liver injury and elevated aminotransferases can occur, Patients with pre-existing liver impairment may be at higher risk, Prior to infusion, assess liver function of all patients by clinical examination and laboratory testing (eg, AST, ALT, total bilirubin, prothrombin time), Administer systemic corticosteroid to all patients before and after onasemnogene abeparvovec infusion, Continue monitoring liver function for at least 3 months after infusion, Acute serious liver injury can occur; before infusion, assess liver function; continue monitoring liver function for at least 3 months after onasemnogene abeparvovec infusion; manage elevated aminotransferase with corticosteroids, Transient decreases in platelet counts, some meeting criteria for thrombocytopenia, observed after infusion; monitor platelet counts before infusion and regularly afterwards (weekly for first month; q2wk for next 2 months), Transient increases in cardiac troponin-I levels observed after infusion; clinical importance of these findings are unknown; cardiac toxicity observed in animal studies; monitor troponin-I before infusion and then for at least 3 months afterwards (weekly for first month; q2wk for next 2 months), There are no data regarding use in pregnant women. 1170572-overview
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ZOLGENSMA ® (onasemnogene abeparvovec-xioi) is a prescription gene therapy used to treat children less than 2 years old with spinal muscular atrophy (SMA). By accessing or using the AdisInsight platform you agree to the terms of use. Federated access using single sign-on credentials. Receiving permission from our users is an important part of our compliance with international privacy regulations. h�tQ�K�@�.���X��A�nE�KA�\]Ă]���6�Co(�n�PAǎ*���s;�qq�H�wh�����{����. We need some information from you before you start using the platform. Reserve your seat now, Adis International Ltd. Part of By clicking send, you acknowledge that you have permission to email the recipient with this information. Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases. Zolgensma ® (onasemnogene abeparvovec-xioi; AVXS-101) is an investigational gene therapy currently in development as a one-time infusion for SMA Type 1. Diseases & Conditions, 2001
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Protecting your personal information is important. Mechanism AVXS‐101 Therapy Increase SMN mRNA and protein from SMN2 gene Strategy Modify SMN2 mRNA splicing to increase amount of functional SMN protein Mechanism SPINRAZA RG7916 branaplam Therapy Loss of motor neurons Prevent motor neuron death Strategy Maintain mitochondria integrity in neurons Mechanism olesoxime Therapy Muscle weakness/ atrophy Increase muscle … Please enter your email address so we can determine if you need to complete a permission form or verify that you have already completed this form. 0
This drug is available at a higher level co-pay. Zolgensma (onasemnogene abeparvovec-xioi), previously known as AVXS-101, is a gene therapy initially developed by AveXis, now part of Novartis, which is further developing and marketing the treatment. endstream
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