What is SPINRAZA (nusinersen)?
SPINRAZA is the first and only FDA-approved treatment for SMA, a genetic disease in which a lack of survival motor neuron (SMN) protein affects the part of the nervous system that controls motor function.
In a controlled study, individuals with infantile-onset SMA treated with SPINRAZA achieved a clinically meaningful improvement in motor function compared to untreated individuals.
In open-label, uncontrolled studies in individuals who had or were likely to develop Type 1, 2, or 3 SMA, some individuals treated with SPINRAZA showed improvements, including:
- The achievement of milestones such as the ability to sit independently, stand, or walk when they would otherwise be unable to do so
- Maintaining milestones at ages when they would be expected to lose them
- Surviving longer than expected considering the typical course of their disease (number of SMN2 copies)
The safety of SPINRAZA has been evaluated in multiple studies of individuals with SMA.
How does SPINRAZA work?
In laboratory tests and animal studies, SPINRAZA was shown to increase full-length SMN protein by targeting the process through which it is produced by a gene called survival motor neuron 2 (SMN2).
How is SPINRAZA administered?
SPINRAZA is given through an established procedure called an intrathecal injection. This delivers medication through the lower back via a lumbar puncture and directly into the central nervous system. This procedure is performed under the direction of healthcare providers experienced in administering lumbar punctures. Sedation may be used depending on the condition of the individual.
The recommended dosage of SPINRAZA is 12 mg (5 mL) per administration. The first 4 doses are called loading doses. The first 3 loading doses should be given at 14-day intervals. The 4th loading dose should be given 30 days after the 3rd dose. A maintenance dose should be given once every 4 months after that.
Your healthcare provider may order laboratory tests before each dose.