What is ZOLGENSMA or onasemnogene abeparvovec-xioi?
ZOLGENSMA is the trade name for onasemnogene abeparvovec-xioi, developed by AveXis, Inc, Novartis Company. ZOLGENSMA is indicated for the treatment of children less than two years of age with SMA– As per the US FDA. This is also the first innovative gene therapy to treat pediatric patients with spinal muscular atrophy, a rare disease and leading genetic cause of infant mortality. SMA is a rare genetic disorder or disease that is induced by a mutation in the survival motor neuron 1 (SMN1) gene. ZOLGENSMA is precisely an adeno-associated virus vector-based gene therapy. It works by targeting the root cause of SMA.
ZOLGENSMA – FDA Approved Cellular and Gene Therapy Product ZOLGENSMA was granted its first US FDA approval in May 2019. This approval was based on its assessment in 36 pediatric patients with infantile-onset SMA. The participants aged of approximately 2 weeks to 8 months at the beginning of study. However, the primary evidence in terms of its effectiveness is based on its assessment in 21 patients in an ongoing clinical trial, at the time of its approval. Patients treated with Zolgensma have demonstrated significant improvement in terms of their ability to achieve developmental motor milestones. ZOLGENSMA received Orphan Drug and Breakthrough Therapy designations by the FDA. More information can be found in the US FDA Press Release.
How does treatment with ZOLGENSMA (onasemnogene abeparvovec-xioi) work?
Cell and Gene Therapies are highly advanced and complex to develop in comparison with routine medications and therapeutic procedures. Some of them are developed specifically for every individual patient through genetic engineering. ZOLGENSMA works by replacin the function of the missing or non working SMN1 gene with a new, working copy of a human SMN gene. ZOLGENSMA is supplied as a suspension meant to administer through intravenous infusion. ZOLGENSMA is provided in a kit containing 2 to 9 vials. Vials are provided in 2 fill volumes: 5.5 mL or 8.3 mL. Intravenous administration of ZOLGENSMA results in cell transduction and expression of the SMN protein, as observed in clinical trials.ZOLGENSMA is delivered as a customized kit. This helps in precisely addressing the dosing requirements for each and every patient receiving the therapy. Refer to the ZOLGENSMA Prescribing Information for specific information.
Patients and caregivers can visit www.zolgensma.com for more specific and comprehensive information. The website has helpful resources for the benefit of Patients. OneGene program is designed to facilitate expert support to the families regarding the treatment and related aspects. The website is meant for US audience.
ZOLGENSMA in India – Is there a possibility?
Information regarding ZOLGENSMA India may be obtained from the regional office of Novartis. Few pediatric patients in India have already received this therapy through special licensing provisions. There may be more channels available to facilitate access to this in the future. You can write to us at nhs@noelgenomics.com and share your interest. We intend to share authentic information and support Indian patients with access to relevant solutions.
