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Biogen wins European spinal muscular atrophy first

Spinraza wins accelerated approval to treat patients with the most common form of SMA


Biogen now has approval in Europe to go with its US license for Spinraza, its drug for the genetic disease spinal muscular atrophy (SMA).

The European Commission gave the go-ahead for Spinraza (nusinersen) as a treatment for patients with the most common form of SMA - known as 5q - which accounts for 95% of all cases of the progressive, debilitating muscle-wasting disease. SMA affects about one in 10,000 live births.

As the first-ever treatment for SMA it was reviewed under the EMA's accelerated assessment programme, intended to expedite access to patients with unmet medical needs.

Spinraza is an antisense oligonucleotide drug licensed by Biogen from Ionis Pharma last year for $75m upfront and $150m in regulatory milestones as well as royalties on sales, and the latter company claims $50m for the EU approval.

The new drug has been predicted to become a blockbuster thanks to its high US price, set at $750,000 in the first year of treatment and dropping to $375,000 thereafter. For now Biogen is keeping its EU pricing and launch plans under wraps, saying availability will vary country by country and it has been "working with health systems and government agencies across the EU to help patients secure access".

In the US sales reached $47m in the first quarter of the year, higher than expected by analysts, which have suggested it could eventually grow into a $1bn-$2bn product.

The wince-inducing price tag led some analysts to suggest Biogen could be dragged into the ongoing debate about the sustainability of pharma pricing in the US, with Leerink saying at the time of Spinraza's approval that it could lead to a payer backlash - some patients with less severe forms of SMA missing out on treatment.

Some US insurers - including Humana and Anthem - implemented restrictions on prescribing earlier this year that reserved the drug for only patients with the most severe (type 1) form of the disease. United Health meanwhile adopted more broad coverage (types 1, 2 and 3) but introduced limits on its use and will only cover beyond a set level of doses if there is clear evidence the drug is working. It doesn’t cover type 4 (late-onset) SMA.

Biogen has been trotting out new data to back up the price tag, including improvements in survival, a reduced need for expensive mechanical ventilation procedures, and showing that its benefits extend to children with less-severe, later-onset forms of SMA.

The biotech needs Spinraza to be a success, as it faces competitive pressure on flagship multiple sclerosis therapy Tecfidera (dimethyl fumarate) and casts around to bulk up what some suggest is a slightly anaemic pipeline. To that end it recently bought late-stage stroke candidate Cirara (glyburide) from Remedy Pharma and an anti-tau antibody from Bristol-Myers Squibb for Alzheimer's disease.

Article by
Phil Taylor

2nd June 2017

From: Regulatory



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