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Date: 2017-01-12

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

Product name: ABO-101

Compound: recombinant adeno-associated viral vector serotype 9 containing the human N-?- acetylglucosaminidase gene

Therapeutic area: Rare diseases - Genetic diseases

Action mechanism:

  • gene therapy. ABO-101 is an adeno-associated viral (AAV)-based gene therapy for Sanfilippo syndrome, which involves a one-time intravenous delivery of a normal copy of the Naglu gene to cells of the central nervous system and peripheral organs with the aim of reversing the effects of the genetic errors that cause the disease.

Company: Abeona Therapeutics (USA - NY) Sermes Planificacion (Spain)

Disease: Sanfilippo syndrome type B

Latest news:

  • • On January 12, 2017, the European Medicines Agency (EMA) Committee for Orphan Medicinal Products has granted Orphan Drug Designation for ABO-101, Abeona's recombinant adeno-associated viral vector serotype 9 containing the human N-?- acetylglucosaminidase gene, for children impacted by Sanfilippo syndrome type B (MPS IIIB).
  • ABO-101 has previously been granted the FDA Orphan Product Designation in April 2014 and received the Rare Pediatric Disease Designation as a pre-requisite part of the FDA’s Priority Review Voucher (PRV) process. The FDA has allowed the Investigational New Drug (IND) for a Phase 1/2 clinical trial, and enrollments are anticipated to begin in the second quarter of 2017.
     

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: 2014-04-30

Orphan status UE: 2017-01-12

Pediatric exclusivit _USA:

Pediatric exclusivity UE:

OTC status:

Other news:

Is general: Yes