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Date: 2016-08-29

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

Product name: recombinant human acid alpha-glucosidase conjugated with mannose-6-phosphate analogues

Compound: recombinant human acid alpha-glucosidase conjugated with mannose-6-phosphate analogues

Therapeutic area: Rare diseases - Genetic diseases

Action mechanism:

enzyme replacement therapy. Nanomedsyn has developed a new enzyme replacement therapy coupled with patented synthetic derivatives called AMFA. These derivatives have been designed for the targeting of a specific membrane lectin, the mannose 6-phosphate receptor, the major addressing pathway to lysosomes. The AMFA compounds have the potential to target various proteins or drugs to tissues and cells expressing these receptors in order to facilitate their cellular entrance and finally lysosomal uptake.

 

Company: NanoMedSyn (France)

Disease:

Pompe's disease

Latest news:

* On 11-13 July, 2016, the Committee for Orphan Medicinal Products (COMP) has recommended the granting of an orphan designation for recombinant human acid alpha-glucosidase conjugated with mannose-6-phosphate analogues for treatment of glycogen storage disease type II (Pompe’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:

Orphan status UE: 2016-08-29

Pediatric exclusivit _USA:

Pediatric exclusivity UE:

OTC status:

Other news:

Is general: Yes