Usual Adult Dose for Spinal Muscular Atrophy

Loading dose: 12 mg intrathecally every 14 days for 3 doses, then 12 mg intrathecally 30 days after the 3rd dose
Maintenance dose: 12 mg intrathecally once every 4 months

Comments:

• For administration, consider sedation; consider ultrasound or other imaging techniques to guide therapy.
• Prior to administration, remove 5 mL from cerebrospinal fluid; administer as an intrathecal bolus injection over 1 to 3 minutes using a spinal anesthesia needle.
• If a loading dose is delayed or missed, administer it as soon as possible, with at least 14-days between doses and continue dosing as prescribed.
• If a maintenance dose is delayed or missed, administer it as soon as possible and continue dosing every 4 months.

Usual Pediatric Dose for Spinal Muscular Atrophy

Loading dose: 12 mg intrathecally every 14 days for 3 doses, then 12 mg intrathecally 30 days after the 3rd dose
Maintenance dose: 12 mg intrathecally once every 4 months

Comments:

• For administration, consider sedation; consider ultrasound or other imaging techniques to guide therapy, especially in younger patients.
• Prior to administration, remove 5 mL from cerebrospinal fluid; administer as an intrathecal bolus injection over 1 to 3 minutes using a spinal anesthesia needle
• If a loading dose is delayed or missed, administer it as soon as possible, with at least 14-days between doses and continue dosing as prescribed.
• If a maintenance dose is delayed or missed, administer it as soon as possible and continue dosing every 4 months.

Renal Dose Adjustments

Use with caution; for urinary protein concentration greater than 0.2 g/L consider repeat testing and further evaluation

• Conduct quantitative spot urine protein testing (preferably with first morning urine specimen) at baseline and prior to each dose

Liver Dose Adjustments

No adjustment recommended

Precautions

CONTRAINDICATIONS:

• None

Dialysis

Data not available

Other Comments

Administration advice:

• For intrathecal use only
• This drug should be administered by, or under the direction of, a healthcare professional experienced in performing lumbar punctures.
• Consider sedation as indicated by the clinical condition of the patient.
• Consider ultrasound or other imaging techniques to guide intrathecal administration, particularly in younger patients.
• Prior to administration, remove 5 mL of cerebrospinal fluid (CSF).
• Administer as an intrathecal bolus injection over 1 to 3 minutes using a spinal anesthesia needle.
• Do not administer in areas of the skin where there are signs of infection or inflammation.

• Protect from light
• Store in the original carton in the refrigerator until time of use; do not freeze.
• May be stored in original carton at or below 30C (86F) for up to 14 days
• Prior to administration, unopened vials may be removed from and returned to the refrigerator, if necessary. If removed from the original carton, the total combined time out of refrigeration should not exceed 30 hours at a temperature that does not exceed 25C (77F)

• Allow vial to warm to room temperature (25C) prior to administration; do not use external heat sources.
• Inspect vial for particulate matter and discoloration prior to administration; do not administer if visible particulates are observed or if the liquid in the vial is discolored.
• The use of external filters is not required.
• Withdraw 12 mg (5 mL) from the single-dose vial and discard unused contents of vial.
• Administer within 4 hours of removal from vial.

• Platelet count; baseline and prior to each dose and as clinically needed
• Prothrombin time; activated partial thromboplastin time; baseline and prior to each dose and as clinically needed
• Quantitative spot urine protein testing, preferably with first morning urine specimen; baseline and prior to each dose
• For urinary protein concentration greater than 0.2 g/L; consider repeat testing and further evaluation

• Patients and caregivers should be instructed to seek medical attention if unexpected bleeding occurs.