Spinraza
Generic name:nusinersen
Dosage form: intrathecal injection
Drug class:Miscellaneous uncategorized agents
What is Spinraza?
Spinraza is a type of medication called an antisense oligonucleotide. It is used to treat children and adults with spinal muscular atrophy (SMA), a type of motor neuron disease. It was the first drug to be approved by the US Food and Drug Administration (FDA) for this group of people.
SMA is an inherited disorder that causes skeletal muscle weakness and wasting, which worsens with age. SMA is most commonly caused by mutations in a gene called SMN1 (survival motor neuron 1), which leaves the body without enough functional SMN protein to keep motor neurons healthy and functioning.
A back-up gene called SMN2 also produces SMN protein, but only a small percentage of the protein produced by SMN2 functions properly. This is because when the protein is being made a portion of the gene called exon 7 tends to be skipped over, so the resulting protein isn't supplied with all of the right building blocks of information it needs to function properly.
Spinraza works by fixing the protein produced by SMN2 so that it functions properly. It does this by making sure that the missing piece of information from exon 7 is included.
Spinraza was approved by the FDA in 2016.
What is Spinraza used for?
Spinraza is a prescription medicine used to treat spinal muscular atrophy (SMA) in pediatric and adult patients.
Important information
Spinraza can cause serious side effects including:
- Increased risk of bleeding. Complications linked to an increased risk of bleeding have been observed after administration of similar medicines. Your healthcare provider will perform blood tests before you start treatment with Spinraza and before each dose to monitor for signs of these risks. Seek medical attention if unexpected bleeding occurs.
- Increased risk of kidney damage, including potentially fatal acute inflammation of the kidney. An increased risk of kidney damage has been observed after administration of similar medicines. Your healthcare provider will perform urine testing before you start treatment with Spinraza and before each dose to monitor for signs of this risk.
What should I tell my doctor before receiving Spinraza?
Before you receive Spinraza, tell your healthcare provider about all of your medical conditions, including if you:
- have a bleeding disorder
- have a kidney disorder
How should I receive Spinraza?
- Spinraza will be administered by a qualified healthcare professional in a treatment center.
- Spinraza is injected into the fluid of the spine (intrathecal injection) over 1 to 3 minutes.
- Before you start treatment with Spinraza and before each dose, your doctor will order blood and urine testing to monitor for the potential risks of bleeding complications or kidney damage.
- You will receive 4 initial loading doses of Spinraza. The first 3 doses will be given at 14-day intervals and the 4th dose wil...
