Version July 2025
Niemann-Pick disease type C:
Note: Use in combination with miglustat. Dose using actual body weight.
Children ≥2 years and Adolescents:
8 to 15 kg: Oral: 47 mg three times daily.
>15 to 30 kg: Oral: 62 mg three times daily.
>30 to 55 kg: Oral: 93 mg three times daily.
>55 kg: Oral: 124 mg three times daily.
Altered kidney function:
Children ≥2 years and Adolescents:
eGFR ≥50 mL/minute: No dosage adjustment necessary.
eGFR 15 to <50 mL/minute:
8 to 15 kg: Oral: 47 mg twice daily.
>15 to 30 kg: Oral: 62 mg twice daily.
>30 to 55 kg: Oral: 93 mg twice daily.
>55 kg: Oral: 124 mg twice daily.
eGFR <15 mL/minute: There are no dosage adjustments provided in the manufacturer's labeling; has not been studied.
Mild to moderate impairment: There are no dosage adjustments provided in the manufacturer's labeling; dosage adjustment likely unnecessary as there were no observed pharmacokinetic differences in patients with mild to moderate liver impairment.
Severe impairment: There are no dosage adjustments provided in the manufacturer's labeling; has not been studied.
(For additional information see "Arimoclomol: Drug information")
Niemann-Pick type C disease: Note: Use in combination with miglustat. Dosing based on actual body weight.
>30 to 55 kg: Oral: 93 mg 3 times daily.
>55 kg: Oral: 124 mg 3 times daily.
eGFR ≥50 mL/minute: No dosage adjustment necessary.
eGFR 15 to <50 mL/minute:
>30 to 55 kg: 93 mg twice daily.
>55 kg: 124 mg twice daily.
eGFR <15 mL/minute: There are no dosage adjustments provided in the manufacturer's labeling (has not been studied).
Mild to moderate impairment (Child-Turcotte-Pugh class A or B): There are no dosage adjustments provided in the manufacturer's labeling; dosage adjustment likely unnecessary as there were no observed pharmacokinetic differences in patients with mild to moderate liver impairment.
Severe impairment (Child-Turcotte-Pugh class C): There are no dosage adjustments provided in the manufacturer's labeling (has not been studied).
The following adverse drug reactions and incidences are derived from product labeling unless otherwise specified. Reported adverse reactions are for adults and pediatrics: may include percentages reported as part of a combination regimen with miglustat.
>10%:
Dermatologic: Urticaria
Endocrine & metabolic: Weight loss
Gastrointestinal: Decreased appetite, diarrhea
Nervous system: Headache, seizure, tremor
Respiratory: Lower respiratory tract infection, upper respiratory tract infection
1% to 10%:
Hematologic & oncologic: Thrombocytopenia
Hypersensitivity: Angioedema
Frequency not defined:
Hematologic & oncologic: Thrombocytopenia
Hypersensitivity: Hypersensitivity reaction
Renal: Increased serum creatinine
There are no contraindications listed in the manufacturer's labeling.
Concerns related to adverse effects:
• Hypersensitivity reactions: Hypersensitivity reactions (eg, urticaria, angioedema) have been reported; reactions occurred within the first 2 months of treatment. Discontinue treatment and treat symptoms immediately in patients with hypersensitivity reactions.
Disease-related concerns:
• Serum creatinine increases: Serum creatinine elevations, typically in the first month of treatment, have occurred; may be due to inhibition of kidney tubular secretion. Increases were not associated with changes in glomerular function and reversed upon discontinuation of therapy.
Excipient information presented when available (limited, particularly for generics); consult specific product labeling.
Capsule, Oral, as citrate:
Miplyffa: 47 mg, 62 mg [contains fd&c blue #1 (brilliant blue)]
Miplyffa: 93 mg, 124 mg
No
Capsules (Miplyffa Oral)
47 mg (per each): $535.80
62 mg (per each): $706.80
93 mg (per each): $1,060.20
124 mg (per each): $1,413.60
Disclaimer: A representative AWP (Average Wholesale Price) price or price range is provided as reference price only. A range is provided when more than one manufacturer's AWP price is available and uses the low and high price reported by the manufacturers to determine the range. The pricing data should be used for benchmarking purposes only, and as such should not be used alone to set or adjudicate any prices for reimbursement or purchasing functions or considered to be an exact price for a single product and/or manufacturer. Medi-Span expressly disclaims all warranties of any kind or nature, whether express or implied, and assumes no liability with respect to accuracy of price or price range data published in its solutions. In no event shall Medi-Span be liable for special, indirect, incidental, or consequential damages arising from use of price or price range data. Pricing data is updated monthly.
Oral: Capsules : Administer with or without food. If patients cannot swallow capsule whole, may open the capsule and sprinkle contents into 15 mL of water or apple juice or sprinkle onto 15 mL of soft food (eg, applesauce, pudding, or yogurt). Administer immediately; do not save mixture for later.
Administration via feeding tube:
Gastric tubes (eg, NG, G-tube): Open capsule, add contents to 20 mL of purified water, and stir the mixture for 15 seconds. Draw up mixture into enteral dosing syringe and immediately administer via feeding tube. After administration, flush feeding tube with 5 mL of purified water.
Oral: Administer with or without food; swallow capsule whole. If patients cannot swallow capsule whole, may open the capsule and sprinkle contents into 15 mL water or apple juice or sprinkle capsule contents onto 15 mL of soft food (eg, applesauce, pudding, yogurt); stir mixture for 15 seconds. Administer immediately; do not save mixture for later use.
Administration via feeding tube:
Gastric tubes (eg, NG, G-tube): Open capsule, add contents to 20 mL of purified water and stir the mixture for 15 seconds; do not mix capsule contents with liquids other than purified water. Draw up mixture into enteral dosing syringe and immediately administer via feeding tube. After administration, flush feeding tube with 5 mL of purified water.
Store at 20°C to 25°C (68°F to 77°F); excursions permitted between 15°C to 30°C (59°F to 86°F). Store in original container. Protect from light.
Treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in combination with miglustat (FDA approved in ages ≥2 years and adults).
Substrate of MATE1/2-K;
Note: Interacting drugs may not be individually listed below if they are part of a group interaction (eg, individual drugs within “CYP3A4 Inducers [Strong]” are NOT listed). For a complete list of drug interactions by individual drug name and detailed management recommendations, use the drug interactions program by clicking on the “Launch drug interactions program” link above.
Note: Interacting drugs may not be individually listed below if they are part of a group interaction (eg, individual drugs within “CYP3A4 Inducers [Strong]” are NOT listed). For a complete list of drug interactions by individual drug name and detailed management recommendations, use the drug interactions program
OCT2 Substrates (Clinically Relevant with Inhibitors): Arimoclomol may increase serum concentration of OCT2 Substrates (Clinically Relevant with Inhibitors). Risk C: Monitor
Consider pregnancy planning and prevention for patients who could become pregnant.
Adverse effects to fertility were observed in animal toxicology studies; data are lacking on possible fertility effects in humans.
Based on data from animal reproduction studies, in utero exposure to arimoclomol may cause fetal harm.
Monitor for signs/symptoms of hypersensitivity, kidney function (non-creatinine based; eg, BUN, cystatin C, or measured GFR).
The mechanism(s) by which arimoclomol exerts its clinical effects in patients with Niemann-Pick disease type C is unknown.
Distribution: Vd: 211 L.
Protein binding: ~10%.
Metabolism: Metabolized predominantly through glutathionation, O-glucuronidation, and NO-oxime cleavage.
Half-life elimination: ~4 hours.
Time to peak: ~0.5 hours.
Excretion: Urine: 77.5% (42% as unchanged drug); feces: ~12%.
Clearance: 34 L/hour.
