Usual Adult Dose for Spinal Muscular Atrophy

5 mg orally once a day

Comments:

• See Other Comments/Administration Advice for important information on dose preparation and administration.

Use: For the treatment of spinal muscular atrophy.

Usual Pediatric Dose for Spinal Muscular Atrophy

2 months to less than 2 years: 0.2 mg/kg orally once a day

2 years and older weight less than 20 kg: 0.25 mg/kg orally once a day

2 years and older weight 20 kg or more: 5 mg orally once a day

Comments:

• See Other Comments/Administration Advice for important information on dose preparation and administration.

Use: For the treatment of spinal muscular atrophy in patients 2 months of age and older.

Renal Dose Adjustments

No adjustment recommended

Liver Dose Adjustments

Mild to moderate hepatic impairment: No adjustment recommended
Severe hepatic impairment: Data not available

Dose Adjustments

Elderly: Clinical studies did not include patients aged 65 years and over to determine if the respond differently; use caution

Precautions

CONTRAINDICATIONS: None

Safety and efficacy have not been established in patients younger than 2 months.

Consult WARNINGS section for additional precautions.

Dialysis

Data not available

Other Comments

Administration advice:

• Take orally once a day after a meal at approximately the same time each day; swallow water after administration to ensure the drug has been completely swallowed
• Oral syringe should be used to measure dose; dose preparation and administration should be demonstrated to patient/caregiver prior to first dose
• Once dose is prepared, it must be administered within 5 minutes; if dose is not taken within 5 minutes, discard dose from the oral syringe and prepare a new dose

INFANTS:

• For infants who are breastfed, administer dose after breastfeeding
• This drug should not be mixed with formula or milk

Nasogastric Tube or Gastrostomy Tube:

• This drug may be administered via NG or G-tube; the tube should be flushed with water after medication has been administered

MISSED DOSE: If a dose is missed, administer as soon as possible if still within 6 hours of the missed dose, and resume regular dosing schedule the next day; otherwise, skip the missed dose and resume regular dosing the next day

VOMITED DOSE or NOT FULLY SWALLOWED: If the full dose is vomited or not fully swallowed, the patient should be instructed to wait until the next day to take their next dose at the regularly scheduled time; do not take another dose to make up for the lost dose

PREPARATION OF ORAL SOLUTION BY PHARMACIST:

• Use caution when handling product; see manufacturer product information for specific reconstitution instructions
• Product should be reconstituted prior to dispensing to patient

Storage requirements:

• Prior to use: Store dry powder in original carton at room temperature (59F to 86F [15C to 30C])
• Once reconstituted: Store in original amber bottle in the refrigerator (36F to 46F [2C too 8C]); stable for 64 days once reconstituted

Patient advice:

• Patients should be instructed to read the US FDA-approved patient labeling (Patient Information and Instructions for Use).
• Patients/caregivers should receive this drug as a liquid solution from the pharmacy; if the bottle is a powder, they should contact their pharmacy for a replacement.
• Women of childbearing potential should be advised on the use of effective contraception during treatment and for 1 month after stopping; women should immediately notify their healthcare provider if they are pregnant or are planning to become pregnant.
• Male patients should understand that their fertility may be compromised while on treatment.