Products

Date: 2018-01-19

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

Product name: AMT-130 -

Compound: adeno-associated viral 5 (AAV5) gene therapy - Adeno-associated viral vector serotype 5 encoding a microRNA targeted to human huntingtin gene

Therapeutic area: Rare diseases - Genetic diseases

Action mechanism:

• gene therapy. AMT-130 consists of an AAV5 vector carrying an engineered micro-RNA specifically designed to silence the huntingtin gene. The therapeutic goal is to inhibit the production of the mutant protein. Using AAV vectors to deliver micro-RNAs directly into the brain represents a highly innovative approach to treating Huntington's disease.

Company: Uniqure (The Netherlands)

Disease: Sanfilippo B syndrome (rare autosomal recessive lysosomal storage disease, which manifests in young children)

Latest news:

• • On January 19, 2018, uniQure  announced that AMT-130, its proprietary gene therapy candidate for Huntington's disease, has received an Orphan Medicinal Product Designation (OMPD) from the European Medicines Agency. AMT-130 represents the first investigational AAV-gene therapy in Huntington's disease to receive such designation.
• • On September 27, 2017, the FDA  granted AMT-130 orphan drug designation 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: 2018-01-19

Pediatric exclusivit _USA:

Pediatric exclusivity UE:

OTC status:

Other news:

Is general: Yes