Products

Date: 2017-09-27

Type of information: Granting of the orphan status in the US

Product name: AMT-130

Compound: adeno-associated viral vector serotype 5 encoding a microRNA targeted to human huntingtin gene

Therapeutic area: Rare diseases - Genetic diseases - Neurodegenerative diseases

Action mechanism:

• gene therapy. AMT-130 consists of an AAV5 vector carrying a DNA cassette encoding artificial micro-RNA (miHTT) that silences the huntingtin gene.

Company: Uniqure (The Netherlands)

Disease: Huntington's disease

Latest news:

• • On September 27, 2017, the FDA  has granted  Orphan Drug Designation to AMT-130 (adeno-associated viral vector serotype 5 encoding a microRNA targeted to human huntingtin gene), for the treatment of Huntington's disease.

 

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: 2017-09-27

Orphan status UE:

Pediatric exclusivit _USA:

Pediatric exclusivity UE:

OTC status:

Other news:

Is general: Yes