Spinraza
Spinraza (nusinersen) is an injectable medication used to treat spinal muscular atrophy (SMA) in children and adults. This medication is injected into the spinal canal by a trained healthcare provider at a treatment facility.
What is Spinraza (nusinersen)?
What is Spinraza (nusinersen) used for?
- Treatment of spinal muscular atrophy (SMA) in children and adults
How Spinraza (nusinersen) works
People with spinal muscular atrophy (SMA) don't have enough of a protein called survival motor neuron (SMN), which is needed for your nerves and muscles to function properly. Spinraza (nusinersen) is an antisense oligonucleotide that helps the body make more SMN proteins to treat people with SMA.
Drug facts
Side effects of Spinraza (nusinersen)
The following side effects may get better over time as your body gets used to the medication. Let your healthcare provider know immediately if you continue to experience these symptoms or if they worsen over time.
Common Side Effects
Infantile-onset SMA (affects infants)
- Lower respiratory infection (55%)
- Constipation (35%)
- Teething (18%)
- Urinary tract infection (9%)
- Stuffy nose (8%)
- Ear infection (6%)
- Passing gas (5%)
- Weight loss (5%)
Later-onset SMA (affects children and adults)
- Fever (43%)
- Headache (29%)
- Vomiting (29%)
- Back pain (25%)
- Nosebleed (7%)
- Falls (5%)
- Stuffy nose (5%)
- Seasonal allergy (5%)
Spinraza (nusinersen) serious side effects
Contact your healthcare provider immediately if you experience any of the following.
- Serious bleeds: bleeding that's difficult to stop, bloody or black-colored stool, vomiting or coughing up blood, dizziness, pale skin
- Kidney damage: low urine amount, swelling in legs or feet, shortness of breath, tiredness, confusion, nausea, weakness, irregular heartbeat, headache, or itchiness
- Atelectasis (collapsed lung): trouble breathing, fast heart rate, cough, chest pain, or skin and lips turning blue
The following Spinraza (nusinersen) side effects have also been reported
Side effects that you should report to your care team as soon as possible:
- Allergic reactions—skin rash, itching, hives, swelling of the face, lips, tongue, or throat
- Bleeding—bloody or black, tar-like stools, vomiting blood or brown material that looks like coffee grounds, red or dark brown urine, small red or purple spots on skin, unusual bruising or bleeding
- Kidney injury—decrease in the amount of urine, swelling of the ankles, hands, or feet
Side effects that usually do not require medical attention (report these to your care team if they continue or are bothersome):
- Back pain
- Constipation
- Fever
- Headache
- Vomiting
Pros and cons of Spinraza (nusinersen)
Pros
- Can be given to both children and adults of all ages
- Maintenance dose only has to be given every 4 months
- Medication gets injected directly into the spinal area, where movement is controlled
Cons
- Must be given by injection, so it might be uncomfortable
- Only given by a trained healthcare provider
- Must complete blood work and urine test before each dose
Pharmacist tips for Spinraza (nusinersen)
- Since Spinraza (nusinersen) can raise the risk of bleeding, it's important to get all of your blood work done so your provider can monitor your bleeding risk. Let your provider know if you experience any unexpected bleeding during your treatment.
- Spinraza (nusinersen) can cause damage to your kidneys. It's important to get your urine tested before each treatment so your provider can monitor your kidney function.
- Let your provider know if you are pregnant, planning to become pregnant, or breastfeeding because it's not known if Spinraza (nusinersen) can harm your baby.
Common questions about Spinraza (nusinersen)
Spinraza (nusinersen) can only be given by a healthcare provider who's trained to inject medications into the spinal canal. You will have to receive Spinraza (nusinersen) injections at a treatment center.
For infantile or early-onset SMA in infants, Spinraza (nusinersen) can help babies achieve milestones, such as head control, rolling, sitting, and standing. For later-onset SMA in children and adults, Spinraza (nusinersen) can help improve walking distance and upper limb function (e.g. arm movements).
Studies showed that people experienced an improvement in their overall motor (movement) function as early as 6 months after starting treatment.
Your provider will request blood work before you start your treatment and before each time you get your Spinraza (nusinersen) injection. Depending on your health conditions, you might need to get blood work more often. It's important to show up to all of your appointments, so speak to your provider to see how often you need to get your labs done.
Contact your provider if you missed your injection apointment, and they will re-schedule you as soon as possible. Your future appointments will have to be rescheduled according to the last injection that you received to make sure there's enough time in between doses.
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Risks and warnings for Spinraza (nusinersen)
Spinraza (nusinersen) can cause some serious health issues. This risk may be even higher for certain groups. If this worries you, talk to your doctor or pharmacist about other options.
Bleeding risk
Spinraza (nusinersen) might raise the risk of bleeding and cause clotting problems, such as thrombocytopenia (low platelet level). Your provider will monitor your platelet levels and bleeding risk before you start your treatment with Spinraza (nusinersen) and before each dose is given. Let your provider know if you have signs and symptoms of bleeding, such as cuts or wounds that bleed for a long time, small red spots under the skin caused by blood leaking from blood vessels, discoloration of the skin caused by bleeding in the skin, nosebleeds, bleeding from your gums, blood in your urine or stool, or heavy menstrual bleeding.
Kidney damage
Spinraza (nusinersen) might raise the risk of kidney damage, including a serious condition called glomerulonephritis. Because Spinraza (nusinersen) gets removed by the kidneys, your provider will check for the health of your kidneys before you start your treatment with Spinraza (nusinersen) and before each dose is given. Let your provider know if you experience signs of kidney damage, such as low urine amount, swelling in your legs or feet, shortness of breath, tiredness, confusion, nausea, weakness, irregular heartbeat, headache, or itchiness while on this medication.
Spinraza (nusinersen) dosage
Typical dosage for Spinraza (nusinersen)
The typical dose is 12 mg (5 mL) injected by intrathecal route (into the spinal canal).
Spinraza (nusinersen) is injected as four loading doses. The first three doses are given every 2 weeks. The fourth dose given 1 month after the third dose. After all loading doses are complete, you'll continue on a maintenance dose given once every 4 months.
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