dm+d

Unassigned

New Medicines

X-linked adrenoleukodystrophy (ALD) in male adults (adrenomyeloneuropathy [AMN] phenotype)

Information

New molecular entity
Minoryx
Minoryx

Development and Regulatory status

Phase III Clinical Trials
Phase III Clinical Trials
Phase III Clinical Trials
Yes
Yes

Category

Selective PPAR gamma agonist with a superior profile for central nervous system-related diseases; a metabolite of pioglitazone.
X-ALD results in cerebral demyelination, axonal dysfunction in the spinal cord leading to spastic paraplegia, adrenal insufficiency & sometimes testicular insufficiency. X-ALD is the most common peroxisomal disorder with an estimated birth incidence of 1/20,000 (male & female). Male hemizygotes may present with neurological symptoms in 2 forms: X-linked cerebral adrenoleukodystrophy (X-CALD) & adrenomyeloneuropathy (AMN). AMN is characterised by the onset of spastic paraplegia in adult men [1].
X-linked adrenoleukodystrophy (ALD) in male adults (adrenomyeloneuropathy [AMN] phenotype)
Oral

Further information

Yes

Evidence based evaluations