The latter recently received a $60 million investment and should be operational in 2020. Zolgensma is meant to halt the progression of the disease by delivering a functional copy of the SMN1 gene, which is defective in SMA patients. News of the delay follows a few months after Zolgensma lost accelerated review status in Europe (see page 5 here). > Catalent Biologics and Novartis’ company AveXis have struck a long-term deal in which the biotech will have dedicated manufacturing space at a Catalent facility for the production of AveXis' Zolgensma, its newly approved gene therapy for spinal muscular atrophy. Bioprocessing begins upstream, most often with culturing of animal or microbial cells in a range of vessel types (such as bags or stirred tanks) using different controlled feeding, aerating, and process strategies. Subscribe to FiercePharma to get industry news and updates delivered to your inbox. However, Narasimhan told analysts this week the EMA and PDMA had extended their deliberations and asked for more information about how Zolgensma is made. The FDA blasted Tennessee compounder Surgery Pharmacy Services for workers' failure to change gloves after removing trash from one of its clean rooms. Scientists at Thomas Jefferson University said they have found a way to train the immune system to tolerate self-antigens that trigger inflammatory responses in MS while leaving the rest of the immune system intact. Nearly five months after snagging a conditional approval for its spinal muscular atrophy (SMA) gene therapy in Europe, Novartis is rolling out fresh data to support a launch that has seen its share of challenges. Of the 33 SMA patients treated so far in the European trial of Zolgensma, 21 had achieved milestones in motor skills during a mean follow-up period of 10.6 months, Novartis said. 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. Of the 33 patients treated so far in the European trial, 21 had achieved milestones in motor skills during a mean follow-up period of 10.6 months that the disease would normally prevent, the company said. Use a + to require a term in results and - to Release. Resolving the queries from the EU required inspections, and it pushed back the decision from the EU’s Committee for Medicinal Products for Human Use (CHMP). All rights reserved. Subscribe to FiercePharma to get industry news and updates delivered to your inbox. And it just revealed new data from a pivotal trial of that drug that intensify Novartis’ marketing challenge. The CMC review team concludes that the manufacturing process for ZOLGENSMA is capable of yielding a product with consistent quality characteristics, and the CMC review team recommends approval. 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 intravenous (IV) infusion. It is most certainly not business as usual at Borderless, however, we consider ourselves fortunate to be able to work without interruption. Reproduction in whole or part is prohibited. Resolving the queries from the EU required inspections, and it pushed back the decision from the EU’s Committee for Medicinal Products for Human Use (CHMP). RELATED: New Zolgensma 'inflection point' is here as Novartis snags EU nod for SMA gene therapy. The Institute for Clinical and Economic Review, a watchdog typically critical of high drug prices, published preliminary evidence in December that showed a one-time gene therapy treatment for SMA set at $2 million could be more cost effective than Spinraza. What Are We Doing Wrong When It Comes to Promoting Women. But then, in October of last year, Novartis was hit with questions from regulators in Europe and Japan about the Zolgensma manufacturing process. That’s because the European approval allows the Zolgensma to be used in children weighing up to 21 kilograms, which basically covers any child under the age of 5. With its new campus (which was previously owned by AstraZeneca), the biotech now runs four U.S. production sites scattered across as many states. Even as it matures, the biopharmaceutical industry is still a highly entrepreneurial one. That’s because the European approval allows the Zolgensma to be used in children weighing up to 21 kilograms, which basically covers any child under the age of 5. With its new campus (which was previously owned by AstraZeneca), the biotech now runs four U.S. production sites scattered across as many states. Meanwhile, Novartis is facing potential competition from more than just Spinraza. Nearly five months after snagging a conditional approval for its spinal muscular atrophy (SMA) gene therapy in Europe, Novartis is rolling out fresh data to support a launch that has seen its share of challenges. That’s a significant difference from the FDA approval, which only covers children under 2 years of age—and it could allow Novartis to offer its therapy to older children currently taking Spinraza. Manufacturing is arguably the largest barrier separating experimental gene therapies from coming to market. However, most media coverage focused on the drug’s $2.1m price tag, setting it in the context of the wider debate about pharmaceutical prices and rare disease therapies. For example, six could sit without assistance for more than 10 seconds and 20 could control their head movements. Most of the children in the study who entered it without requiring ventilation remained free of ventilator support, and 67% were able to eat without help, Novartis said during the World Muscle Society 2020 Virtual Congress.
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