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Biogen bounces back with Spinraza early treatment data

Takes battle to Novartis in youngest patients


Biogen’s spinal muscular atrophy treatment (SMA) Spinraza is facing a major threat from Novartis’ newly-approved gene therapy Zolgensma, and needs strong data to help it stand its ground and maintain sales growth.

Yesterday the US biotech company unveiled some unexpectedly compelling data in an early setting, showing the drug could help prevent the onset of the muscle-wasting disease in infants before any symptoms begin to show – and keep children alive who would otherwise have died before their second birthday or require permanent ventilation.

The new data takes the fight to Zolgensma, which Novartis has gained US approval for only in infants with SMA under two years of age.

Biogen's NURTURE trial is an ongoing phase 2, open-label study of 25 pre-symptomatic patients with SMA (most likely to develop SMA Type 1 or 2) who received their first dose of Spinraza before six weeks old, and produced unparalleled results in comparison to the natural history of SMA.

As of March 2019, 100% were alive without a need for permanent ventilation, with the median age of the study participants at nearly three years old.

This is remarkable as majority of patients with SMA type 1 who go untreated will not reach their second birthday without permanent ventilation.

What’s more all of the children were sitting independently, and the majority (88%) were walking, many of them within the normal timeframe for a toddler.

Biogen says Spinraza has demonstrated longer term efficacy up to nearly 4 years, with participants continuing to make progress and showing no signs of loss of motor function. It also says no new safety concerns were identified over this period, with the drug well tolerated.

This may be one area in which Biogen will seek to contrast its treatment with Zolgensma – Novartis’ gene therapy, which saw a death of one patient in clinical trials.

Nevertheless, Biogen’s new data shows striking similarities to Novartis’ pivotal study for Zolgensma.

Novartis’ gene therapy aims to address SMA's underlying issue, effectively replacing the missing or defective SMN1 gene with a functional copy that makes SMN protein, improving motor neuron function and survival.

Its recent US approval was based on the START phase 1 study, which saw 15 patients infused with Zolgensma alive at 24 months without the need for permanent ventilation.

Additionally, 92% of patients who received the proposed therapeutic dose of Zolgensma could sit unassisted for five seconds or more, a milestone never achieved in the natural history of SMA Type 1.

Novartis also said those who voluntarily enrolled in an ongoing observational follow-up of the trial have maintained their developmental motor milestones, including patients who are four years post-infusion.

Competition in SMA will further intensify with the anticipated future approval of Roche’s contender, risdiplam.

Analysts at Jefferies recently forecast that the three therapies would split the market into equal thirds, with Zolgensma claiming newly-diagnosed patients, with Spinraza and Roche’s drug used in older patients with more advanced cases of the disease.

However the new NURTURE data challenges that assumption, and sets up a pitched battle between Biogen and Novartis to win the right to treat these small numbers of patients.

“A few years ago, SMA patients had no treatment options and faced significant care challenges,” said Kenneth Hobby, President of Cure SMA, a patient advocacy organisation dedicated to the treatment and cure of SMA. “However, the future of SMA has changed and especially with early treatment patients now have a very strong chance to reach age appropriate developmental milestones.

He added: “These new data demonstrate the dramatic impact where children are now walking independently at four years of age, when the usual lifespan would be under two if untreated. This study provides additional evidence on the maintenance of these improvements. It’s critical that research in SMA continues to support the generation of real-world evidence in patients of all ages so that we better understand the long-term implications of SMA and treatment across all types.”

In addition to the evolving data, another key dynamic in the market will be price and payment options.

Spinraza has been on the US market since 2016, and comes in at a list price $750,000 for the initial year and $375,000 per year thereafter.

Biogen’s drug has to be administered lifelong, in contrast to Zolgensma, which is a one-time infusion of gene therapy. Novartis has set its price at a record-breaking $2.12m per patient, but argues that this works out considerably cheaper than a lifetime of Spinraza therapy.

Article by
Andrew McConaghie

2nd July 2019

From: Marketing



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