Date: 2016-03-21
Type of information: Granting of the orphan status in the EU
Product name: BMN 270 - valoctocogene roxaparvovec- adeno-associated viral vector serotype 5 containing a B-domain deleted variant of human coagulation factor VIII gene
Compound: valoctocogene roxaparvovec- adeno-associated viral vector serotype 5 containing a B-domain deleted variant of human coagulation factor VIII gene
Therapeutic area: Rare diseases - Genetic diseases - Hematological diseases
Action mechanism:
- gene therapy. BMN 270 is an AAV-factor VIII vector, designed to restore factor VIII plasma concentrations, essential for blood clotting in patients with hemophilia A. The gene therapy program for Hemophilia A was originally licensed from University College London and St. Jude Children's Research Hospital in February 2013 and has since been developed at BioMarin's facilities.
Company: Biomarin Pharmaceutical (USA - CA)
Disease: hemophilia A
Latest news:
- • On March 24, 2016, BioMarin Pharmaceutical announced that BMN 270 has been granted orphan drug designation by the European Commission. BioMarin is currently conducting a Phase 1/2 study to evaluate the safety and efficacy of BMN 270 gene therapy in patients with severe hemophilia A.
- • On February 29, 2016, the FDA has granted orphan drug status for adeno-associated viral vector serotype 5 containing a B-domain deleted variant of human coagulation factor VIII gene for treatment of hemophilia A.
- • On February 25, 2016, the Committee for Orphan Medicinal Products (COMP) has recommended the granting of an orphan designation for adeno-associated viral vector serotype 5 containing a B-domain deleted variant of human coagulation factor VIII gene for treatment of hemophilia A.
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: 2016-02-29
Orphan status UE: 2016-03-21
Pediatric exclusivit _USA:
Pediatric exclusivity UE:
OTC status:
Other news:
- • On October 18, 2017, BioMarin Pharmaceutical provided an update on its development portfolio. The company announced that Good Manufacturing Practices (GMP) production of BMN 270 has commenced at its gene therapy manufacturing facilities in Novato. This production will support clinical development activities and anticipated commercial demand. This facility is capable of supporting approximately 2,000 patients per year, and the production process was developed in accordance with International Conference on Harmonisation guidance for Pharmaceuticals for Human Use facilitating worldwide registration with health authorities.
Is general: Yes
