What is Spinraza™?
Spinraza™ (nusinersen) is the first treatment approved to treat SMA in Europe.
Spinraza™ is intrathecal applied medicine that is designed to increase SMN protein levels in the central nervous system (CNS). This SMN-enhancing therapy is a antisense oligonucleotide that targets the SMN2 gene and helps to increase the amount of stable SMN protein made by this gene.
At a glance
- Spinraza is approved for all ages and types of SMA in many European countries
- Access to Spinraza varies
- Spinraza is given via an intrathecal (IT) injection, which is an injection directly into the cerebrospinal fluid through the lower back. Individuals receive four “loading doses” within the first 2 months of treatment. Once those loading doses are completed, they receive a maintenance dose every 4 months for the duration of the individual’s life.
- Spinraza is an antisense oligonucleotide.
- Spinraza was tested in multi-centre clinical trials in a number of infants and children.
- After the pre-clinical work and early clinical testing was complete, Ionis Pharmaceuticals partnered with Biogen to test Spinraza in clinical trials and bring it to the markets.