Date: 2014-01-22
Type of information: Granting of the orphan status in the EU
Product name: MIN-101 - pioglitazone
Compound: pioglitazone
Therapeutic area: Rare diseases
Action mechanism: Pioglitazone selectively stimulates nuclear receptor peroxisone proliferator-activated receptor gamma (PPAR-gamma). It modulates the transcription of the insulin-sensitive genes involved in the control of glucose and lipid metabolism in the lipidic, muscular tissues and in the liver. Proof of concept for the efficacy of Pioglitazone was provided by the results of biochemical, neuropathological and motor effects in two mouse models of ALD (which are deficient in ABCD1 and ABCD2). In such models, Pioglitazone halted the progression of the disease (in terms of movement disorder and nerve damage), reversed bioenergetic failure and prevented oxidative stress These results have been published in Brain and highlighted by Nature Reviews of Neurology.
Company: Minoryx Therapeutics (Spain)
Disease: adrenoleukodystrophy
Latest news: * On 7-9 January 2014, the Committee for Orphan Medicinal Products (COMP) has recommended the granting of an orphan designation for pioglitazone for treatment of adrenoleukodystrophy.
Patents:
Submission of marketing authorization application USA :
Submission of marketing authorization application UE:
Withdrawal of marketing authorization application USA:
Withdrawal of marketing authorization application UE:
US authorization:
UE authorization:
Favourable opinion UE:
Favourable opinion USA:
Orphan status USA:
Orphan status UE: 2014-02-19
Pediatric exclusivit _USA:
Pediatric exclusivity UE: OTC status: Other news:
