ArimoclomolApplication Scope

Arimoclomol is indicated for use in combination with miglustat for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients 2 years of age and older.

Arimoclomol Characteristics

Ingredients: Each capsule contains arimoclomol citrate as the active ingredient.
Properties: Arimoclomol functions as a heat shock protein amplifier, enhancing the production of molecular chaperones that assist in the proper folding and function of proteins.
Packaging Specification: Available in capsule form with the following strengths and corresponding capsule colors:
- 47 mg: White opaque body with black printing “47”, green opaque cap with black printing “OZ”
- 62 mg: White opaque body with black printing “62”, yellow opaque cap with black printing “OZ”
- 93 mg: White opaque body with black printing “93”, orange opaque cap with black printing “OZ”
- 124 mg: White opaque body with black printing “124”, red opaque cap with black printing “OZ”
  Each strength is supplied in bottles of 90 capsules.
Storage: Store at 20°C to 25°C (68°F to 77°F); excursions permitted between 15°C and 30°C (59°F to 86°F). Keep in the original container and protect from light.
Expiry Date: Refer to the expiration date printed on the packaging.
Executive Standard: Not specified in the provided sources.
Approval Number: Not specified in the provided sources.
Date of Revision: September 2024.
Manufacturer:
- Manufactured by: Zevra Denmark A/S, 2000 Frederiksberg, Denmark
- Manufactured for: Zevra Therapeutics, Inc., Celebration, FL 34747, USA

Guidelines for the Use of Arimoclomol

Dosage and Administration:
- Adults and Pediatric Patients (≥2 years):
  - 8 to 15 kg: 47 mg orally three times daily
  - 15 to 30 kg: 62 mg orally three times daily
  - 30 to 55 kg: 93 mg orally three times daily
  - 55 kg: 124 mg orally three times daily
    Administer with or without food.
- Patients with Renal Impairment (eGFR ≥15 to <50 mL/min):
  - 8 to 15 kg: 47 mg orally twice daily
  - 15 to 30 kg: 62 mg orally twice daily
  - 30 to 55 kg: 93 mg orally twice daily
  - 55 kg: 124 mg orally twice daily
    No dosage adjustment is recommended for patients with eGFR ≥50 mL/min.
- Administration Instructions:
  - Swallow capsules whole.
  - For patients unable to swallow capsules:
    - Open the capsule and sprinkle the entire contents into 15 mL of water or apple juice, or mix with 15 mL of soft food (e.g., applesauce, pudding, yogurt).
    - Stir the mixture for 15 seconds and consume immediately.
  - For administration via feeding tube:
    - Open the capsule and sprinkle the contents into 20 mL of water.
    - Stir for 15 seconds and administer immediately through the feeding tube.
    - Flush the tube with 5 mL of water after administration.
Adverse Reactions:
- Common adverse reactions (≥15%):
  - Upper respiratory tract infection
  - Diarrhea
  - Decreased weight
- Hypersensitivity reactions, such as urticaria and angioedema, have been reported. Discontinue use if severe reactions occur.
Contraindications:
- None identified.
Precautions:
- Use with caution in patients with renal impairment; monitor renal function.
- Potential for embryofetal toxicity; advise pregnant females of potential risks.
- Safety and efficacy have not been established in patients younger than 2 years.

Arimoclomol Interactions

Drug Interactions:
- Arimoclomol is an inhibitor of the organic cation transporter 2 (OCT2).
- Coadministration with OCT2 substrates (e.g., metformin, dalfampridine, dofetilide) may increase plasma concentrations of these drugs, potentially leading to adverse effects. Monitor patients closely and adjust dosages as necessary.

Indications: Treatment of neurological manifestations of Niemann-Pick disease type C in combination with miglustat.

Note:

If there is a new packaging for the drug, the new packaging shall prevail. The above information is sourced from DengYue Medicine.
It is only for internal discussion among medical staff and does not serve as a basis for medication. For specific medication guidelines, please consult the attending physician.