EMA gives positive opinion on Biogen’s spinal muscular atrophy drug Spinraza

Biogen has revealed that Spinraza (nusinersen), its treatment for spinal muscular atrophy (SMA) , has received a positive opinion from the EMA’s Committee for Medicinal Products for Human Use (CHMP).

Biogen licensed Spinraza from US firm Ionis Pharmaceuticals, and the drug made the news in November last year when it reached its primary endpoint in Phase 3 trials. It later made headlines when it was approved by the FDA in January – not because it had earned the authorisation, but because of its price tag: Spinraza entered the US market at $125,000 per dose. The drug requires up to six doses in the first year of treatment, and it is necessary to use it indefinitely, meaning huge costs for the average patient. Despite this, Biogen claimed that insurers had met the decision with positive reactions.

Spinraza was reviewed under the EMA’s accelerated assessment programme, and it has now been given the go-ahead for use in treating 5q SMA, the most common form of the debilitating illness, accounting for 95% of cases.  

The decision was based on the fact that patients treated with the drug in clinical trials were enabled to kick, roll, sit and crawl among other activities compared to those untreated. The drug also reduced the risk of death or permanent ventilation, while maintaining a favourable safety profile.

"The positive CHMP opinion, which was expedited under the accelerated assessment programme, recognises the compelling efficacy profile of Spinraza and underscores the significant unmet need for an effective SMA treatment in Europe," said Michael Ehlers, Executive Vice President, Research and Development at Biogen. "We look forward to the European Commission’s decision and believe Spinraza has the potential to make a meaningful impact for individuals with SMA in the EU."