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New data revealed for Novartis SMA gene therapy
Novartis has revealed new data for its one-time gene therapy for spinal muscular atrophy (SMA) in older children.
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Zolgensma (onasemnogene abeparvovec)
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Year in review: EPR’s top stories of 2023
EPR wraps up the year with a selection of top stories from 2023, highlighting key trends and topics such as environmental monitoring (EM), quality (QA/QC), manufacturing, regulation and clinical development.
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Realising milestones with gene therapy for SMA
Novartis Gene Therapies' Vice President for Clinical Development & Analytics shares what shaped the success of one of the few commercially-available gene therapies.
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Realising the potential of AAV gene therapies
In this article, Dr Rajiv Vaidya, Head of Manufacturing Science & Technology at Andelyn Biosciences, explores strategies for improving the scalability and cost‑effectiveness of AAV production while maintaining regulatory compliance.
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Novartis releases long-term data for SMA gene therapy
Long-term follow-up study data for Zolgensma®, Novartis’ one-time gene therapy for spinal muscular atrophy (SMA), has shown promising overall milestone achievements.
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Final positive results of Zolgensma® trial for SMA published
After previous reports that nearly all spinal muscular atrophy (SMA) patients treated with Zolgensma® achieved key motor milestones, Novartis announces publication of full SPR1NT results.
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Challenges in downstream purification of advanced therapies
John Liddell, Chief Technologist at the Centre for Process Innovation (CPI), writes about the challenges associated with gene therapy products, which constitute a major portion of the overall cell and gene therapy market.
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Zolgensma® meaningfully improves spinal muscular atrophy in patients
Asymptomatic and symptomatic children treated with Zolgensma® (onasemnogene abeparvovec) gene therapy achieved motor milestones never observed in the natural history of spinal muscular atrophy.
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NICE approves gene therapy for spinal muscular atrophy
Zolgensma, a one-off, potentially curative treatment for type 1 spinal muscular atrophy, is set to become the most expensive drug ever approved for use on the UK’s NHS.
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EMA’s transformative treatments of 2020
In this article, we summarise the trial findings for the European Medicines Agency’s 13 most transformative therapies of 2020.
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February Pharmacovigilance Risk Assessment Committee (PRAC) meeting highlights
The EMA’s Pharmacovigilance Risk Assessment Committee (PRAC) began two new reviews and wrote guidance for healthcare professionals on the potential side effects of Strimvelis, Venclyxto and Zolgensma.
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SMA drug Zolgensma® has “significant therapeutic benefit”, according to interim data
Treatment with Zolgensma® (onasemnogene abeparvovec) resulted in rapid and sustained improvement in motor function in spinal muscular atrophy (SMA) patients.
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Committee for Medicinal Products for Human Use (CHMP) meeting highlights
A summary of the CHMP meeting conclusions, including medicines recommended for approval and indication extension and several safety review findings.
