Spinraza (nusinersen), a treatment for most forms of spinal muscular atrophy (SMA), took a major step forward to becoming available across the European Union, with the Committee for Medicinal Products for Human Use (CHMP) recommending its approval. CHMP is a branch of the European Medicines Agency (EMA), the equivalent of the U.S. Food and Drug Administration for Europe.
The recommendation was based on positive results seen in two Phase 3 clinical trials, ENDEAR and CHERISH. The CHMP recommendation now goes to the European Commission, and if accepted, Spinraza will become first approved SMA treatment in Europe.
The FDA approved Spinraza to treat SMA types 1-3 in children and adults in December, making it the first approved treatment for the disease in the U.S.
Spinal muscular atrophy involves the loss of motor neurons (nerve cells that control muscle movement) in the spinal cord and lower brain stem, resulting in severe and progressive muscular atrophy (muscle wasting) and weakness. The most severe type of SMA, type 1 or infantile-onset, can cause paralysis and difficulty performing basic functions, like breathing and swallowing.
In the ENDEAR (infantile-onset SMA) trial (NCT02193074), a greater number of infants with SMA type 1 treated with Spinraza achieved a motor milestone — defined as an ability to move muscles in line with their developmental age — compared to untreated infants. Responses included the ability to kick, head control, rolling, sitting, and crawling. A lower risk of death or difficulty breathing was also seen in treated babies.
In the CHERISH trial (NCT02292537), improvement was seen in motor function in SMA type 2 children (those with later-onset SMA) treated with Spinraza compared to the placebo group. Improvements were measured by the Hammersmith Functional Motor Scale Expanded (HFMSE), which is used to assess motor function in SMA patients.
Spinraza has been proven to be safe. It is delivered by lumbar puncture, a medical procedure in which a needle is inserted into the spinal canal, and is administered by healthcare professionals experienced with lumbar punctures due to the risks associated with this procedure (headache, backpain, vomiting).
“The positive CHMP opinion … recognizes the compelling efficacy profile of SPINRAZA and underscores the significant unmet need for an effective SMA treatment in Europe,” Michael Ehlers, executive vice president of Research and Development at Biogen, said in a press release. “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.”
A European Commission decision is expected in a few months.