Search Results for: zOLGENSMA

Novartis CGTs first quarter 2022: Zolgensma up, Kymriah down

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Novartis reports increased sales for gene therapy Zolgensma due to expanded global access, but the failure in the second-line DLBCL setting will affect Kymriah sales. For Swiss Pharma giant Novartis’ cell and gene therapy (CGT) division, first quarter 2022 results were mixed. On the positive side, Zolgensma (onasemnogene abeparvovec) pulled in $363 million during the quarter, up 18% year-on-year due to increased access outside of the US. Sales of the one-time spinal muscular atrophy (SMA) gene therapy — approved in the…

April 27, 2022

Novartis’s $115m pays off as FDA approves second Zolgensma site

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The Novartis facility in North Carolina will begin shipping Zolgensma gene therapy doses immediately after receiving US FDA commercial licensure approval. The Swiss Big Pharma firm announced yesterday the US Food and Drug Administration (FDA) has granted approval to its multi-product gene therapy manufacturing facility in Durham, North Carolina. The 170,000 square-foot facility site will primarily be used to produce Zolgensma (onasemnogene abeparvovec. Novartis added Zolgensma to its pipeline through the $8.7 billion acquisition of AveXis in May 2018 and…

April 6, 2022

Novartis close to EU approval for Zolgensma at last

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Novartis’s AveXis unit has received a CHMP nod for the spinal muscular atrophy gene therapy at its latest meeting, sending it on the final steps to market in the EU. After several delays plus a row over a controversial lottery-style managed access program in Europe, Novartis gene therapy Zolgensma (onasemnogene abeparvovec) has finally been granted a positive opinion by the EU’s Committee for Medicinal Products for Human Use (CHMP). The committee has recommended a conditional marketing authorization of Zolgensma for the…

April 6, 2020

Novartis opens its largest gene therapy plant to support Zolgensma

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The doors have opened at a facility in Longmont, Colorado nine months after Novartis acquired it to support its gene therapy business unit AveXis. In April 2019, Novartis bought the Longmont facility from fellow Big Biopharma player AstraZeneca in efforts to grow out its gene therapy production network. Nine months on and the Swiss Biopharma has confirmed the site is open and prepped to support its AveXis unit in the production of therapies including Zolgensma (onasemnogene abeparvovec-xioi1), which was approved…

January 10, 2020

Zolgensma: Manufacturing questions delay decision in EU and Japan

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Novartis has asked the EMA to inspect its Zolgensma plant in San DIego after the agency – and Japan’s PDMA – delayed approval decisions and asked for more details of how the gene therapy is made. Zolgensma is a gene therapy. It is designed to halt progressive spinal muscular atrophy (SMA) – motor neurone loss and muscle wasting – in kids with a defective version of a gene called SMN1. The drug encodes a functional copy of the gene enabling sustained…

October 25, 2019

Zolgensma data: ‘Heavy-handiness’ of FDA a warning to industry, not just Novartis

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The US FDA may have made an example of Novartis in the way it handled the revelation of Zolgensma data manipulation, says the Alliance for Regenerative Medicine. A month after the US Food and Drug Administration (FDA) approved the one-time gene therapy Zolgensma (onasemnogene abeparvovec), marketing authorization holder Novartis alerted the agency to a data manipulation issue that impacted the accuracy of certain data from product testing performed in animals submitted in the biologics license application (BLA). In its response,…

September 29, 2019

FDA and Novartis back Zolgensma amid data manipulation issues

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The US FDA remains confident that Zolgensma will not be pulled after being alerted to allegations of data manipulation by the gene therapy manufacturer AveXis. In May 2019, Novartis company AveXis received US Food and Drug Administration (FDA) approval for its $2.1 million single-dose, one-time gene therapy Zolgensma (onasemnogene abeparvovec) to treat children less than two years of age with spinal muscular atrophy (SMA). A month later, AveXis informed the Agency “about a data manipulation issue that impacts the accuracy…

August 7, 2019

Zolgensma: We all know the price, but how is it made?

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Making Zolgensma, the new “world’s most expensive drug,†is a month-long process that depends on vector tech licensed from Regenxbio. Zolgensma is a gene therapy. It is designed to halt progressive spinal muscular atrophy (SMA) – motor neurone loss and muscle wasting – in people who have a defective version of the gene SMN1. The drug addresses the underlying causes of SMA according to Novartis spokesman Eric Althoff. He told us, “Zolgensma provides a functional copy of the human SMN…

June 10, 2019

Zolgensma approval marks success for REGENXBIO’s vector platform

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REGENXBIO could receive up to $80 million after the US FDA approved Novartis/AveXis’ gene therapy Zolgensma (onasemnogene abeparvovec), which uses the firm’s NAV Technology Platform. Last week, the US Food and Drug Administration (FDA) approved Zolgensma, a single-dose gene therapy developed by Novartis acquisition AveXis, for the treatment of children less than two years of age with spinal muscular atrophy (SMA). The approval triggered a $3.5 million (€3.1 million) milestone payment to Maryland-based biotech REGENXBIO. Zolgensma is based on REGENXBIO’s…

May 29, 2019

Novartis wins approval for $2.1m gene therapy Zolgensma

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Novartis says it is ready to launch Zolgensma (onasemnogene abeparvovec) within the next few weeks after receiving FDA approval for its spinal muscular atrophy (SMA) single-dose gene therapy. Zolgensma, a single-dose, one-time gene therapy has been approved to treat children less than two years of age with spinal muscular atrophy (SMA). “Today’s approval marks another milestone in the transformational power of gene and cell therapies to treat a wide range of diseases,†acting US Food and Drug Administration (FDA) commissioner…

May 27, 2019