September 3, 2020

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In our quest to find new medicines, we consistently rank among the world’s top companies investing in research and development. Patients with pre existing liver impairment may be at higher risk of experiencing serious liver injury. Delay ZOLGENSMA infusion until full-term gestational age is reached. Novartis unveiled interim phase 3 data on its gene therapy for spinal muscular atrophy, Zolgensma, which showed significant gains in motor skills … 2007;22:1027–49.7. Zolgensma® (onasemnogene abeparvovec-xioi), Adeno-associated virus vector-based gene therapy. The first form is a more severe variant, causing rapid motor neuron loss and resulting in death or permanent ventilation support requirement in more than 90% of patients if left untreated. The FDA approved the drug in May 2019. Sign up to follow @Novartis at https://twitter.com/novartisnewsFor Novartis multimedia content, please visit https://www.novartis.com/news/media-libraryFor questions about the site or required registration, please contact [email protected]. Find out more at https://www.novartis.com. The Zolgensma drug will be available in single-use vials, each containing a nominal concentration of 2.0 × 1013 vector genomes (vg) per millilitre for intravenous infusion. More than 600 patients have been treated with Zolgensma, including clinical trials, commercially and through the managed access program. “Patients with SMA now have another treatment option to minimize the progression of SMA and improve survival. Novartis and the investigator considered the events and death to be unrelated to treatment with Zolgensma based on autopsy findings. STR1VE-EU is distinct in its inclusion and exclusion criteria and baseline clinical characteristics of enrolled patients compared with START or STR1VE-US. Such forward-looking statements are based on our current beliefs and expectations regarding future events, and are subject to significant known and unknown risks and uncertainties. A one-time intravenous administration of Zolgensma results in expression of the SMN protein in a child’s motor neurons, which improves muscle movement and function, and survival of a child with SMA. Even as it matures, the biopharmaceutical industry is still a highly entrepreneurial one. 2002;125:1624–34.6. Making Zolgensma, the new “world’s most expensive drug,” is a month-long process that depends on vector tech licensed from Regenxbio. SMA is a rare, genetic neuromuscular disease caused by a lack of a functional SMN1 gene, resulting in the rapid and irreversible loss of motor neurons, affecting muscle functions, including breathing, swallowing and basic movement. The approved drug, Zolgensma, is an AAV-based gene therapy first developed by AveXis, Inc. and acquired by Novartis in April 2018 for $8.7 billion. Zolgensma is approved in the US, Japan and, most recently, Brazil, for patients with SMA under the age of two. Novartis products reach nearly 800 million people globally and we are finding innovative ways to expand access to our latest treatments. Beyond Zolgensma, there are conventional therapies in late stage development by Roche (Risdiplam) and an additional program by Novartis (Branaplam). Zolgensma is indicated for the treatment of children less than two years of age with SMA. Drug products combine active pharmaceutical ingredients with excipients in a final formulation for delivery to patients in liquid or lyophilized (freeze-dried) packaged forms — with the latter requiring reconstitution in the clinical setting. As a result of the Zolgensma case, it is expected that the FDA will become more rigorous in their review of applications and demand more data to support approvals. Trial design and other details are being evaluated and a comprehensive update on the overall Novartis SMA clinical development program will be provided at a future time following further discussions with health authorities. Specifically, some patients in STR1VE-EU had a more severe disease phenotype at baseline, including lower CHOP-INTEND scores and the need for nutritional and ventilatory support. Novartis Institutes for BioMedical Research, https://www.novartis.com/news/media-library. The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The vector delivers a fully functional copy of human SMN gene into the target motor neuron cells. The most common side effects of Zolgensma are elevated liver enzymes and vomiting. Out of the 21 patients, 19 no longer needed permanent ventilation and 47.6% were able to sit without support for at least 30 seconds. The disease is caused by a genetic defect in the survival motor neuron (SMN) gene, which encodes the SMN protein essential for the survival of motor neurons. Zolgensma ® is designed to address the genetic root cause of SMA by providing a functional copy of the human SMN gene to halt disease progression through sustained SMN protein expression with a single, one-time IV infusion. Priced at $2.1m for five years, the drug is one of the most expensive drugs to be approved. Contact us for more information about DeciBio’s 2019 NGS Report, Competitive Intelligence (CI) tools, or other market intelligence products. 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