Arimoclomol 47 mg, 62mg, 93mg Suppliers & Bulk Manufacturers

Available Forms: Capsules

Available Strengths: 47 mg, 62mg, 93mg

Reference Brands: MIPLYFFA (USA)

Category: Orphan Drugs

Arimoclomol (brand name Miplyffa) is an orphan drug used for treating the neurological manifestations of Niemann-Pick disease type C (NPC) in patients aged 2 years and older. It works by enhancing heat-shock protein production, helping protect cells from stress and improving protein handling. Arimoclomol is available as oral capsules in multiple strengths and is used together with miglustat to slow neurological progression in NPC.

Product Description:

Arimoclomol is a prescription medication indicated for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) when used in combination with miglustat in adults and pediatric patients aged 2 years and older. NPC is a rare, progressive genetic lysosomal storage disorder characterized by impaired lipid trafficking in cells, which leads to lipid accumulation in the brain and other organs, resulting in severe neurological and systemic symptoms. Arimoclomol works by enhancing the cellular heat shock response—boosting production of protective heat shock proteins (HSPs) that help maintain normal protein folding and assist in cellular stress management. It is orally administered in capsule form in weight-based doses, usually taken three times a day, and may be swallowed whole or opened and mixed with liquid or soft foods for patients with swallowing difficulties. Arimoclomol is notable for its ability to cross the blood–brain barrier, a critical feature for therapies targeting neurodegenerative processes. Common side effects include upper respiratory tract infection, diarrhea, and decreased weight. Arimoclomol was approved by the U.S. Food and Drug Administration in 2024 and represents a first-in-class treatment for NPC.