First Patient Treated with Zolgensma in UK

Date: June 1, 2021 | Country: UNITED KINGDOM | Region: EUROPE | Type: Other |
Keywords: #atrophy #baby #genetherapy #muscular #novartis #patient #sma #spinal #zolgensma

PRICENTRIC BRIEF:

  • A five-month-old has become the first patient to be treated with Novartis’ Zolgensma (onasemnogene abeparvovec) in the UK, following the closing of a “landmark” deal for the therapy in March
  • The recipient, Arthur Morgan, was born six weeks premature in December and ultimately underwent the gene therapy infusion at the end of May after being diagnosed with spinal muscular atrophy (SMA) less than three weeks earlier
  • NHS England and Novartis signed an agreement in March to make Zolgensma available for SMA type 1 patients on the UK’s NHS

THE DETAILS

LONDON, United Kingdom – A five-month-old has become the first patient to be treated with Novartis’ Zolgensma (onasemnogene abeparvovec) in the UK, following the closing of a “landmark” deal for the therapy in March.

The recipient, Arthur Morgan, was born six weeks premature in December and ultimately underwent the gene therapy infusion at the end of May after being diagnosed with spinal muscular atrophy (SMA) less than three weeks earlier.

He received the dose at Evelina London Children’s Hospital on 25 May.

NHS England and Novartis signed an agreement in March to make Zolgensma available for SMA type 1 patients on the UK’s NHS.

The one-time gene therapy can help babies to reach milestones such as breathe without a ventilator, sit up on their own and move and walk after a single injection, but has a £1.79 million per dose price tag.

However, the two parties struck a deal to secure the innovative gene therapy at a “substantial” confidential discount.

In the document, the organization determined that because of the uncertainty in the clinical data, the cost-effectiveness estimates for Zolgensma treating type 1 SMA are uncertain. However, they are likely to be within a range of what NICE considers an effective use of NHS resources for highly specialized technologies.

Further, the consultation clarified that because of the limited trial data for children aged 7 to 12 months, their treatment should be discussed by a national multidisciplinary team.

Up until now, NICE has recommended the disease-modifying therapy Spinraza (nusinsersen) for some people with pre-symptomatic SMA and types 1, 2 or 3 SMA as part of a managed access agreement.

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