Zolgensma (onasemnogene abeparvovec)

news

Novartis releases long-term data for SMA gene therapy

20 March 2023 | By Catherine Eckford (European Pharmaceutical Review)

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

20 June 2022 | By Mandy Parrett (European Pharmaceutical Review)

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

22 February 2022 | By John Liddell (CPI)

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

21 June 2021 | By Hannah Balfour (European Pharmaceutical Review)

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

9 March 2021 | By Hannah Balfour (European Pharmaceutical Review)

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

24 February 2021 | By Hannah Balfour (European Pharmaceutical Review)

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

15 February 2021 | By Hannah Balfour (European Pharmaceutical Review)

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

5 October 2020 | By Hannah Balfour (European Pharmaceutical Review)

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

30 March 2020 | By Hannah Balfour (European Pharmaceutical Review)

A summary of the CHMP meeting conclusions, including medicines recommended for approval and indication extension and several safety review findings.