BPL announces strong progress on the world’s first licensed Factor X product
London, UK – Bio Products Laboratory (BPL) is pleased to announce continued progress on what will be the world’s first licensed Factor X concentrate. The submission is ground-breaking; BPL’s Factor X is the first pure factor X concentrate developed globally, and also the first treatment specifically for the ultra-orphan congenital Factor X deficiency.
- Biologics License Application (BLA) submitted to the FDA
- FDA inspection completed
- Efficacy data to be presented at WFH 2014
Following the successful completion of a Phase III study recruiting patients from the US, Spain, Germany, Turkey and the UK, BPL submitted a BLA for Factor X to the US Food and Drug Administration (FDA) in July 2013. Given its orphan drug status, the FDA has granted BPL a priority review, which if positive could deliver an approval in the first half of 2014.
“Given the significant unmet need associated with Factor X deficiency and the low prevalence of this condition, the clinical data were encouraging, and used for a BLA submission,” said Tim Aldwinckle, Medical Director for BPL.
Another important milestone was reached this October, as BPL hosted inspectors from the FDA. The visit was a Pre-Approval Inspection for Factor X, to support BPL’s BLA submission to allow sales of Factor X in the US. The inspection took place from 21-25 October 2013, and was a critical step on the path to licensing.
BPL has chosen the upcoming World Federation of Hemophilia (WFH) congress for the release of the Factor X safety and efficacy clinical study data. BPL will share data from two studies covering on-demand treatment in Factor X deficient adults, as well as prophylactic use in such patients undergoing surgery. The WFH World Congress takes place from 11-15 May 2014 in Melbourne, Australia, and is the ideal launch meeting for Factor X given that more than 4000 hemophilia stakeholders from 120 countries will be in attendance.
Kim Bjoernstrup, PRUK CEO comments:
“I am extremely pleased with the continued progress we are making towards the launch of the world’s first licensed Factor X product, and we believe this will be an important advance for patients and their carers. This is the first of many important initiatives BPL is undertaking, and BPL will continue to pursue development opportunities such as a 10% IVIG formulation, a second Factor X indication for the reversal of Factor Xa inhibition, and registration of albumin in China. We will pursue some of these projects in-house, and for some specific projects we would seek to collaborate with suitable partners.”