Biogen today announced the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion recommending the granting of a marketing authorization for SPINRAZA (nusinersen) to treat patients with spinal muscular atrophy (SMA). The CHMP reviewed SPINRAZA under an accelerated assessment program, which is a regulatory mechanism to facilitate earlier access to patients for medicines that fulfill unmet medical needs. SPINRAZA is the first treatment for SMA to be recommended by the CHMP for approval in the European Union (EU).
"The positive CHMP opinion, which was expedited under the accelerated assessment program, recognizes the compelling efficacy profile of SPINRAZA and underscores the significant unmet need for an effective SMA treatment in Europe," said Michael Ehlers, M.D., Ph.D., 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."
The CHMP positive opinion is now referred to the European Commission (EC), which grants the marketing authorization for centrally authorized medicines in the EU. The CHMP recommended an indication for the treatment of 5q SMA, which refers to the most common form of the disease and represents approximately 95% of all SMA cases. A decision from the EC is expected in the next few months.
Data Supporting Positive Opinion
The recommendation was primarily based on the CHMP's assessment of two pivotal controlled studies, ENDEAR (infantile-onset SMA) and CHERISH (later-onset SMA), which both demonstrated the clinically meaningful efficacy and favorable safety profile of SPINRAZA.
- In ENDEAR, a statistically significant greater percentage of individuals with infantile-onset SMA (most likely to develop Type 1) treated with SPINRAZA achieved a motor milestone response compared to untreated individuals. Motor milestones achieved in some individuals treated with SPINRAZA included the ability to kick, head control, rolling, sitting and crawling. In addition, a statistically significant reduction in the risk of death or permanent ventilation was seen in SPINRAZA-treated individuals compared to untreated individuals at the end of study analysis.
- In CHERISH, there was a statistically significant and clinically meaningful improvement in motor function in individuals treated with SPINRAZA with later-onset SMA (most likely to develop Type 2 or Type 3) compared to untreated individuals at the interim analysis. Improvements were measured by the Hammersmith Functional Motor Scale Expanded (HFMSE). The HFMSE is a reliable and validated tool specifically designed to assess motor function in children with SMA.
- Data from open-label studies in pre-symptomatic and symptomatic individuals most likely to develop Type 1, 2 or 3 were consistent with the results of the pivotal studies and were considered supportive of the recommended indication. The overall findings of these studies support the efficacy and safety of SPINRAZA in individuals with SMA.
- SPINRAZA demonstrated a favorable safety profile. SPINRAZA is to be administered by healthcare professionals experienced in doing lumbar punctures due to the risks associated with this procedure (e.g. headache, backpain, vomiting).
SPINRAZA Program Status
Biogen licensed the global rights to develop, manufacture and commercialize SPINRAZA from Ionis Pharmaceuticals (NASDAQ: IONS), a leader in antisense therapeutics. Biogen and Ionis conducted an innovative clinical development program that moved SPINRAZA from its first dose in humans in 2011 to its first regulatory approval by the United States Food and Drug Administration (FDA) in 2016.
SPINRAZA was first approved by the FDA on December 23, 2016 within three months of regulatory filing. Biogen has also submitted regulatory filings in Japan, Canada, Australia and Switzerland and plans to initiate additional filings in other countries in 2017.