Under threat from Novartis, Biogen hails new data from SMA drug Spinraza

Under threat from a gene therapy from Novartis, Biogen is making the case for its drug Spinraza for the muscle wasting disease spinal muscular atrophy, with new data that it hopes persuades doctors to prescribe it.

Despite its steep list price of around £450,000 for the first year of treatment NHS England and NICE have struck a deal with Biogen that will allow patients access under a “managed access agreement”.

This involves a confidential price cut and funds treatment for a time-limited period until further data is available on Spinraza’s effectiveness.

The latest data from the NURTURE study could well help NICE with its decisions about how to fund Spinraza in the longer term.

Results in NURTURE involve data from the longest study SMA in pre-symptomatic infants, involving up to 45.1 months of analysis.

Data demonstrate efficacy and safety in patients treated pre-symptomatically in comparison with the natural history of the disease.

The 25 patients treated with Spinraza had continuous improvement with the overwhelming majority of patients achieving motor milestones in a normal timeframe.

At data cut off in March this year, 100% of the patients were alive without need for permanent ventilation.

The median age of participants was nearly three years old, and the majority of untreated patients with SMA type 1 never reach their second birthday without permanent ventilation.

All of the infants were sitting independently, in comparison to the natural history of this disease where no patients with SMA type 1 would be able to do so and patients with SMA type 2 would need assistance.

In the study group 88% of the infants were walking independently with many of them doing so in the normal timeframe for a toddler. In the natural history of SMA, patients with SMA Type 1 or Type 2 are never able to walk independently.

They were also approaching the maximum mean score on a measure of motor function in SMA, and Spinraza demonstrated longer term efficacy up to nearly four years, with participants continuing to make progress and showing no signs of loss of motor function. No new safety concerns emerged or were identified.

Under the managed access agreement NICE targeted the sickest babies with type 1 disease, before moving on to babies, children and young adults with less severe type 2 and type 3 disease once services were established.

Just how this data in presymptomatic children will affect arrangements remains to be seen in the UK, and in the US where payers may be charged the full list price.

Darryl De Vivo, Sidney Carter professor of neurology and paediatrics at the Columbia Irving Medical Center in New York City, said: “These study results demonstrate the durable impact of pre-symptomatic, proactive treatment on transforming the natural course of this disease. We are seeing an extensive number of patients continually meeting child motor development milestones and making unprecedented gains in a previously hopeless and often fatal condition.

“Spinraza is setting patients on a path toward survival, greater mobility and independence, which is helping improve outcomes for patients of all ages.”

Spinraza is an anti-sense drug that works by preventing RNA coding for the mutated proteins that cause SMA.

But in May the FDA approved Novartis’ rival SMA gene therapy Zolgensma (onasemnogene abeparvovec-xioi), which is based around a single shot that corrects the disease at a genetic level.

Zolgensma costs around $2.125 million, which works out at $425,000 per year over five years, around half the cost of ten years’ treatment with Spinraza.