Spinraza 12mg/5ml Inj, 5ml
It is used to treat spinal muscular atrophy.
Drug Class
Drug for Spinal Muscular Atrophy (SMA)
Pharmacologic Class
Antisense oligonucleotide (ASO); Survival Motor Neuron 2 (SMN2) splicing modifier
Pregnancy Category
Not available
FDA Approved
Dec 2016
DEA Schedule
Not Controlled
Overview
What is this medicine?
Spinraza is a medicine given by injection into the fluid around the spinal cord. It helps people with spinal muscular atrophy (SMA) make more of a protein called SMN, which is important for muscle strength and movement. By increasing this protein, Spinraza can help improve or stabilize muscle function in SMA patients.
How to Use This Medicine
To use this medication correctly, follow your doctor's instructions and carefully read all accompanying information. Adhere to the dosage and administration guidelines provided. This medication is administered via injection into the spine.
If you need to store this medication at home, consult with your doctor, nurse, or pharmacist to determine the appropriate storage method.
In the event that you miss a dose, contact your doctor promptly to receive guidance on the next steps to take.
If you need to store this medication at home, consult with your doctor, nurse, or pharmacist to determine the appropriate storage method.
In the event that you miss a dose, contact your doctor promptly to receive guidance on the next steps to take.
Lifestyle & Tips
- Continue with regular physical therapy, occupational therapy, and respiratory support as recommended by your healthcare team.
- Maintain regular follow-up appointments for injections and monitoring.
- Report any new or worsening symptoms promptly to your doctor.
Available Forms & Alternatives
Available Strengths:
Dosing & Administration
Adult Dosing
Standard Dose:
Loading Doses: 12 mg (5 mL) administered by intrathecal injection on Days 0, 14, 28, and 63. Maintenance Doses: 12 mg (5 mL) administered by intrathecal injection once every 4 months thereafter.
Dose Range:
12 - 12 mg
Pediatric Dosing
Neonatal:
Loading Doses: 12 mg (5 mL) administered by intrathecal injection on Days 0, 14, 28, and 63. Maintenance Doses: 12 mg (5 mL) administered by intrathecal injection once every 4 months thereafter.
Infant:
Loading Doses: 12 mg (5 mL) administered by intrathecal injection on Days 0, 14, 28, and 63. Maintenance Doses: 12 mg (5 mL) administered by intrathecal injection once every 4 months thereafter.
Child:
Loading Doses: 12 mg (5 mL) administered by intrathecal injection on Days 0, 14, 28, and 63. Maintenance Doses: 12 mg (5 mL) administered by intrathecal injection once every 4 months thereafter.
Adolescent:
Loading Doses: 12 mg (5 mL) administered by intrathecal injection on Days 0, 14, 28, and 63. Maintenance Doses: 12 mg (5 mL) administered by intrathecal injection once every 4 months thereafter.
Dose Adjustments
Renal Impairment:
Mild:
No dose adjustment recommended due to minimal systemic exposure.
Moderate:
No dose adjustment recommended due to minimal systemic exposure.
Severe:
No dose adjustment recommended due to minimal systemic exposure.
Dialysis:
No specific recommendations; minimal systemic exposure suggests no adjustment needed.
Hepatic Impairment:
Mild:
No dose adjustment recommended due to minimal systemic exposure and metabolism primarily by exonuclease-mediated hydrolysis.
Moderate:
No dose adjustment recommended due to minimal systemic exposure and metabolism primarily by exonuclease-mediated hydrolysis.
Severe:
No dose adjustment recommended due to minimal systemic exposure and metabolism primarily by exonuclease-mediated hydrolysis.
Pharmacology
Mechanism of Action
Nusinersen is an antisense oligonucleotide (ASO) designed to increase the production of full-length survival motor neuron (SMN) protein. It targets the SMN2 pre-mRNA, promoting the inclusion of exon 7 during splicing, which is typically excluded in patients with spinal muscular atrophy (SMA). This leads to the production of functional SMN protein, which is deficient in SMA patients.
Pharmacokinetics
Absorption:
Bioavailability:
Not applicable (intrathecal administration)
Tmax:
1-6 hours (in CSF)
FoodEffect:
Not applicable (intrathecal administration)
Distribution:
Vd:
Approximately 1.5 L (in CSF)
ProteinBinding:
Low
CnssPenetration:
Direct (intrathecal administration)
Elimination:
HalfLife:
Approximately 135 to 177 days (in CSF)
Clearance:
Not well-defined for systemic clearance due to minimal systemic exposure; primarily cleared from CSF via bulk flow and enzymatic degradation.
ExcretionRoute:
Not fully characterized; metabolites primarily eliminated via hydrolysis.
Unchanged:
Not applicable (primarily metabolized by hydrolysis)
Pharmacodynamics
OnsetOfAction:
Clinical improvement observed within months of initiation.
PeakEffect:
Continues to improve over time with maintenance doses.
DurationOfAction:
Long-acting due to slow elimination from the CNS (CSF half-life of several months).
Safety & Warnings
Side Effects
Urgent Side Effects: Seek Medical Help Right Away
While rare, some people may experience severe and potentially life-threatening side effects when taking this medication. If you notice any of the following symptoms, contact your doctor or seek immediate medical attention:
Signs of an allergic reaction, such as:
+ Rash
+ Hives
+ Itching
+ Red, swollen, blistered, or peeling skin with or without fever
+ Wheezing
+ Tightness in the chest or throat
+ Trouble breathing, swallowing, or talking
+ Unusual hoarseness
+ Swelling of the mouth, face, lips, tongue, or throat
Signs of bleeding, including:
+ Vomiting or coughing up blood
+ Vomit that resembles coffee grounds
+ Blood in the urine
+ Black, red, or tarry stools
+ Bleeding from the gums
+ Abnormal vaginal bleeding
+ Unexplained bruises or bruises that increase in size
+ Uncontrollable bleeding
Signs of kidney problems, such as:
+ Inability to urinate
+ Changes in urine output
+ Blood in the urine
+ Sudden significant weight gain
Signs of a urinary tract infection (UTI), including:
+ Blood in the urine
+ Burning or pain while urinating
+ Frequent or urgent need to urinate
+ Fever
+ Lower abdominal pain
+ Pelvic pain
Chills
Change in sputum color
Shortness of breath
Cough
Ear pain
Falls
Other Possible Side Effects
Like all medications, this drug can cause side effects. However, many people experience no side effects or only mild ones. If you encounter any of the following side effects or any other symptoms that concern you or do not resolve, contact your doctor or seek medical attention:
Signs of a common cold
Constipation
Headache
Back pain
Gas
Weight loss
* Nausea and vomiting
Reporting Side Effects
This list is not exhaustive, and you may experience other side effects. If you have questions or concerns about side effects, consult your doctor. You can also report side effects to the FDA at 1-800-332-1088 or online at https://www.fda.gov/medwatch.
While rare, some people may experience severe and potentially life-threatening side effects when taking this medication. If you notice any of the following symptoms, contact your doctor or seek immediate medical attention:
Signs of an allergic reaction, such as:
+ Rash
+ Hives
+ Itching
+ Red, swollen, blistered, or peeling skin with or without fever
+ Wheezing
+ Tightness in the chest or throat
+ Trouble breathing, swallowing, or talking
+ Unusual hoarseness
+ Swelling of the mouth, face, lips, tongue, or throat
Signs of bleeding, including:
+ Vomiting or coughing up blood
+ Vomit that resembles coffee grounds
+ Blood in the urine
+ Black, red, or tarry stools
+ Bleeding from the gums
+ Abnormal vaginal bleeding
+ Unexplained bruises or bruises that increase in size
+ Uncontrollable bleeding
Signs of kidney problems, such as:
+ Inability to urinate
+ Changes in urine output
+ Blood in the urine
+ Sudden significant weight gain
Signs of a urinary tract infection (UTI), including:
+ Blood in the urine
+ Burning or pain while urinating
+ Frequent or urgent need to urinate
+ Fever
+ Lower abdominal pain
+ Pelvic pain
Chills
Change in sputum color
Shortness of breath
Cough
Ear pain
Falls
Other Possible Side Effects
Like all medications, this drug can cause side effects. However, many people experience no side effects or only mild ones. If you encounter any of the following side effects or any other symptoms that concern you or do not resolve, contact your doctor or seek medical attention:
Signs of a common cold
Constipation
Headache
Back pain
Gas
Weight loss
* Nausea and vomiting
Reporting Side Effects
This list is not exhaustive, and you may experience other side effects. If you have questions or concerns about side effects, consult your doctor. You can also report side effects to the FDA at 1-800-332-1088 or online at https://www.fda.gov/medwatch.
Seek Immediate Medical Attention If You Experience:
- Severe headache, nausea, vomiting, vision changes, or altered mental status (could indicate hydrocephalus)
- Unusual bleeding or bruising, nosebleeds, blood in urine or stool, or prolonged bleeding from minor cuts (could indicate problems with blood clotting or platelets)
- Swelling in the legs or ankles, decreased urination, or unusual fatigue (could indicate kidney problems)
- Any signs of infection at the injection site (e.g., redness, swelling, pain, fever)
Before Using This Medicine
Before Taking This Medication: Important Information to Share with Your Doctor
It is essential to inform your doctor about the following:
Any allergies you have, including allergies to this medication, its components, or other substances, such as foods or drugs. Be sure to describe the symptoms you experienced as a result of the allergy.
All medications you are currently taking, including prescription and over-the-counter (OTC) drugs, natural products, and vitamins. This information will help your doctor and pharmacist assess potential interactions between this medication and other substances you are taking.
* Any existing health problems, as this medication may interact with certain conditions.
To ensure your safety, it is crucial to verify that it is safe to take this medication with all your other medications and health conditions. Never start, stop, or change the dose of any medication without first consulting your doctor.
It is essential to inform your doctor about the following:
Any allergies you have, including allergies to this medication, its components, or other substances, such as foods or drugs. Be sure to describe the symptoms you experienced as a result of the allergy.
All medications you are currently taking, including prescription and over-the-counter (OTC) drugs, natural products, and vitamins. This information will help your doctor and pharmacist assess potential interactions between this medication and other substances you are taking.
* Any existing health problems, as this medication may interact with certain conditions.
To ensure your safety, it is crucial to verify that it is safe to take this medication with all your other medications and health conditions. Never start, stop, or change the dose of any medication without first consulting your doctor.
Precautions & Cautions
It is essential to inform all your healthcare providers, including doctors, nurses, pharmacists, and dentists, that you are taking this medication.
Regular blood work and laboratory tests should be conducted as directed by your doctor to monitor your condition.
You may experience an increased risk of bleeding, so it is crucial to exercise caution and avoid injuries. To minimize this risk, use a soft-bristled toothbrush and an electric razor for shaving.
There is a potential risk of kidney problems associated with this type of medication, which in some cases can be fatal. Discuss this risk with your doctor to understand the implications.
If you are pregnant, planning to become pregnant, or are breast-feeding, you must consult your doctor to weigh the benefits and risks of this medication to both you and your baby.
Regular blood work and laboratory tests should be conducted as directed by your doctor to monitor your condition.
You may experience an increased risk of bleeding, so it is crucial to exercise caution and avoid injuries. To minimize this risk, use a soft-bristled toothbrush and an electric razor for shaving.
There is a potential risk of kidney problems associated with this type of medication, which in some cases can be fatal. Discuss this risk with your doctor to understand the implications.
If you are pregnant, planning to become pregnant, or are breast-feeding, you must consult your doctor to weigh the benefits and risks of this medication to both you and your baby.
Overdose Information
Overdose Symptoms:
- Symptoms of overdose are not well-established due to the route of administration and limited systemic exposure. Potential symptoms could include severe neurological effects if excessive amounts were administered intrathecally.
What to Do:
In case of suspected overdose, seek immediate medical attention. Management is supportive. Call 1-800-222-1222 (Poison Control).
Drug Interactions
Monitoring
Baseline Monitoring
Platelet count
Rationale: Risk of thrombocytopenia.
Timing: Prior to first dose and prior to each subsequent dose.
Coagulation parameters (PT/aPTT)
Rationale: Risk of coagulopathy.
Timing: Prior to first dose and prior to each subsequent dose.
Renal function (serum creatinine, BUN, urinalysis)
Rationale: Risk of glomerulonephritis.
Timing: Prior to first dose and periodically thereafter.
Routine Monitoring
Platelet count
Frequency: Prior to each dose
Target: Normal laboratory range
Action Threshold: Clinically significant decrease or below normal range; consider delaying or discontinuing treatment.
Coagulation parameters (PT/aPTT)
Frequency: Prior to each dose
Target: Normal laboratory range
Action Threshold: Clinically significant prolongation or outside normal range; consider delaying or discontinuing treatment.
Renal function (serum creatinine, BUN, urinalysis)
Frequency: Periodically
Target: Normal laboratory range
Action Threshold: Clinically significant increase in creatinine/BUN or new onset proteinuria; consider further evaluation and potential treatment modification.
Symptom Monitoring
- Signs and symptoms of hydrocephalus (e.g., severe headache, nausea, vomiting, papilledema, altered mental status)
- Unusual bleeding or bruising (e.g., petechiae, purpura, epistaxis, hematuria)
- Signs of renal dysfunction (e.g., edema, decreased urine output, fatigue)
Special Patient Groups
Pregnancy
Limited human data on nusinersen use in pregnant women are insufficient to inform a drug-associated risk for major birth defects or miscarriage. Animal studies showed no adverse developmental effects. Due to minimal systemic exposure, the risk to the fetus is considered low, but use only if the potential benefit justifies the potential risk to the fetus.
Trimester-Specific Risks:
First Trimester:
Risk not specifically defined by trimester due to limited data and low systemic exposure. Consider potential benefits vs. risks.
Second Trimester:
Risk not specifically defined by trimester due to limited data and low systemic exposure. Consider potential benefits vs. risks.
Third Trimester:
Risk not specifically defined by trimester due to limited data and low systemic exposure. Consider potential benefits vs. risks.
Lactation
There are no data on the presence of nusinersen in human milk, the effects on the breastfed infant, or the effects on milk production. Due to minimal systemic exposure, excretion into human milk is expected to be low. Consider the developmental and health benefits of breastfeeding along with the mother’s clinical need for Spinraza and any potential adverse effects on the breastfed infant from Spinraza or from the underlying maternal condition.
Infant Risk:
Unknown, likely low due to minimal systemic exposure.
Pediatric Use
Spinraza is approved for the treatment of spinal muscular atrophy in pediatric patients of all ages, including infants. The dosing regimen is the same for pediatric and adult patients. Special considerations include the need for sedation for intrathecal administration in younger children and infants.
Geriatric Use
Clinical studies of Spinraza did not include sufficient numbers of patients aged 65 and over to determine whether they respond differently from younger patients. No specific dose adjustment is recommended for geriatric patients.
Clinical Information
Clinical Pearls
- Spinraza is administered via intrathecal injection, requiring a healthcare professional experienced in performing lumbar punctures.
- Patients, especially infants and young children, may require sedation for the intrathecal injection procedure.
- Careful monitoring of platelet counts, coagulation parameters (PT/aPTT), and renal function (serum creatinine, BUN, urinalysis) is crucial before each dose and periodically.
- Patients should be monitored for signs and symptoms of hydrocephalus, bleeding, and renal toxicity.
- Spinraza is a long-term treatment; adherence to the maintenance dosing schedule is important for sustained efficacy.
Alternative Therapies
- Onasemnogene abeparvovec-xioi (Zolgensma): A gene therapy for SMA, administered as a one-time intravenous infusion.
- Risdiplam (Evrysdi): An oral small molecule splicing modifier for SMA.
Cost & Coverage
Average Cost:
Very high (e.g., hundreds of thousands of USD per year) per year
Insurance Coverage:
Specialty tier; typically requires prior authorization and may be subject to strict coverage criteria due to high cost and specific indication.
General Drug Facts
If your symptoms or health problems persist or worsen, it is essential to contact your doctor promptly. To ensure safe use, never share your medication with others or take someone else's medication. Store all medications in a secure location, out of reach of children and pets, to prevent accidental ingestion. Dispose of unused or expired medications properly; do not flush them down the toilet or pour them down the drain unless instructed to do so by a healthcare professional or pharmacist. If you are unsure about the proper disposal method, consult your pharmacist, as they can provide guidance on disposal procedures and potential drug take-back programs in your area. Additionally, some medications may have a separate patient information leaflet; check with your pharmacist for more information. If you have any questions or concerns about your medication, discuss them with your doctor, nurse, pharmacist, or other healthcare provider. In the event of a suspected overdose, immediately contact your local poison control center or seek emergency medical attention. Be prepared to provide information about the medication taken, the amount, and the time it was taken to ensure timely and effective treatment.