SMA treatment receives positive CHMP opinion

The CHMP has given a positive opinion recommending that Biogen’s treatment for patients with spinal muscular atrophy (SMA), SPINRAZA (nusinersen), be granted marketing authorisation.

SPINRAZA was reviewed by the CHMP under an accelerated assessment programme, which is used by the committee to facilitate earlier access to patients for medicines that fulfil unmet medical needs. It is the first treatment for SMA recommended by CHMP for approval in the EU.

The recommendation was based on two controlled studies that both demonstrated the clinical efficacy and favourable safety profile of the treatment (ENDEAR and CHERISH). Its recommendation is as an indication for the treatment of 5q SMA, which is the most common form of the disease. This positive opinion will be reviewed by the European Commission, which grants marketing authorisation for medicines in the EU.

Dr Michael Ehlers, PhD, executive vice president, research and development at Biogen, said: “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. 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.”

SPINRASA is an antisense oligonucleotide designed to treat SMA caused by mutations or deletions in the SMN1 gene located in chromosome 5q that leads to survival motor neuron protein deficiency. It is administered through intrathecal injection, which delivers therapies directly to the cerebrospinal fluid around the spinal cord.

Biogen, a biotechnology company, focuses on the discovery, development and delivery of therapies for neurological and neurodegenerative diseases.