SMA Europe is delighted to report that Biogen’s marketing authorisation application for Nusinersen as a Treatment for SMA has been validated by EMA . This means that EMA will start to review the application.
Biogen today announced that its marketing authorisation application (MAA) for Nusinersen, an investigational treatment for SMA, has been validated by the European Medicines Agency (EMA) and the priority review for New Drug Application (NDA) has been accepted by the U.S. Food and Drug Administration (FDA).
Nusinersen had previously been granted Accelerated Assessment status by the EMA’s Committee for Medicinal Products for Human Use (CHMP). The regulatory review process for these applications has now been initiated in the U.S. and EU. Both the Priority Review and Accelerated Assessment designations can reduce the standard review time. If approved, Nusinersen would be the first therapy for SMA, a leading genetic cause of infant mortality.
Biogen intends to market Nusinersen under the brand name SPINRAZA. This name has been conditionally accepted by the FDA and the CHMP and will be confirmed upon approval.
A reminder of how Nusinersen works
People living with SMA have a fault in a gene called SMN1, so that the protein it codes for, SMN Protein, is not produced. The lack of this protein causes damage to the nerve cells (motor neurons) which control muscle activity. Due to the damage to these cells, the muscles are not asked to work so they shrink (atrophy).
However, humans have a back-up copy of this SMN gene, which is called SMN2. Unfortunately, SMN2 only produces ~15% functional SMN protein, which is not enough to compensate for the lack of SMN protein produced by the SMN1 gene.
Nusinersen is an oligonucleotide (a molecule) which allows this back-up gene, SMN2, to produce a greater amount of functional SMN protein.
Please read the full press release.
Biogen press release – 28th October 2016