Arimoclomol Citrate — Active Pharmaceutical Ingredient

Arimoclomol Citrate (1 brand name, 1 dosage form, 1 route)

Available dosage forms: CAPSULE.

Routes of administration: ORAL.

Brand Names

• MIPLYFFA

Indications and Usage

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

Contraindications

None.

Adverse Reactions

The following clinically significant adverse reactions are described below and elsewhere in the labeling: Hypersensitivity Reactions [see Warning and Precautions (5.1)]. Increased Creatinine without Affecting Glomerular Function [see Warnings and Precautions (5.3)].

Mechanism of Action

The mechanism(s) by which arimoclomol exerts its clinical effects in patients with NPC is unknown.