Zolgensma (onasemnogene abeparvovec)

news

New data revealed for Novartis SMA gene therapy

4 March 2024 | By Catherine Eckford (European Pharmaceutical Review)

Novartis has revealed new data for its one-time gene therapy for spinal muscular atrophy (SMA) in older children.

article

Year in review: EPR’s top stories of 2023

18 December 2023 | By Catherine Eckford (European Pharmaceutical Review)

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.

article

Realising milestones with gene therapy for SMA

11 May 2023 | By Catherine Eckford (European Pharmaceutical Review), Dr Sitra Tauscher-Wisniewski (Novartis Gene Therapies)

Novartis Gene Therapies' Vice President for Clinical Development & Analytics shares what shaped the success of one of the few commercially-available gene therapies.

article

Realising the potential of AAV gene therapies

27 April 2023 | By Dr Rajiv Vaidya (Andelyn Biosciences)

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.

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.

news

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.

article

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.

news

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.

news

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.

article

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.

news

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.

news

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.

news

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.