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BPL bags FDA approval for rare bleeding disorder therapy

First drug approved for hereditary Factor X deficiency
FDA

UK firm Bio Products Laboratory (BPL) has won US approval for Coagadex, the first drug approved specifically for patients with the rare bleeding disorder hereditary Factor X deficiency.

The new therapy is a plasma-derived Factor X that has been approved by the FDA for on-demand control, of bleeding episodes in patients aged over 12, as well as preventative use in patients with a mild form of the condition who need to undergo surgery.

Similar to haemophilia, Factor X deficiency is an inherited disorder - affecting men and women equally - where the blood does not clot as it should. It affects around 1 in 500,000 people, with the number of US patients estimated at 400-600.

The US regulator notes that treatment is usually based on giving fresh plasma or plasma-derived prothrombin complex concentrates to stop or prevent bleeding.

"The approval of Coagadex is a significant advancement for patients who suffer from this rare but serious disease," commented Karen Midthun, who heads the FDA's Center for Biologics Evaluation and Research.

Approval of Coagadex was based on an open-label trial in 16 patients with moderate or severe Factor X deficiency, with the drug effectively controlling bleeding episodes -often with just one dose - and achieving a quick recovery rate. It also demonstrated a half-life in the body of around 30 hours, allowing for flexible dosing around surgeries.

There were 187 bleeding episodes reported in the study, with 91% of patients reporting the treatment as 'excellent', and another 7.5% rating it as 'good'.

BPL has also submitted a marketing authorisation application to the European Medicines Agency (EMA) that is also currently under review.

The company is also in the process of carrying out a study of Coagadex as a prophylactic treatment for children aged under 12 which is due to complete next summer.

Article by
Phil Taylor

21st October 2015

From: Regulatory

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