US FDA to review CSL haemophilia treatment

The US Food and Drug Administration has accepted for review CSL's licence application for its latest treatment for haemophilia.

US drug regulators will review CSL's latest treatment for haemophilia, after a clinical trial showed promising results.

CSL says the US Food and Drug Administration has accepted for review CSL's licence application for its recombinant single-chain factor VIII treatment for haemophilia A.

Haemophilia is a potentially life-threatening blood clotting disorder where there is insufficient clotting factor VIII or IX in the blood to help control bleeding.

People affected by haemophilia can experience prolonged or spontaneous bleeding in the muscles, joints or internal organs.

The condition affects more than 175,000 people worldwide, most of whom have haemophilia A.

CSL chief scientific officer Andrew Cuthbertson said CSL has the only recombinant single-chain factor VIII product in late-stage development for the management of haemophilia A.

"We are excited to be one step closer to providing this innovative treatment to patients in the US," Dr Cuthbertson said in a statement on Tuesday.

CSL's recombinant single-chain factor VIII treatment is designed to provide greater molecular stability.

It uses a covalent bond that forms one structural entity - a single chain - to improve the stability of factor VIII and provide longer-lasting factor VIII activity.

Patients may also require fewer doses of the treatment.

In the first half of the 2014/15 financial year, haemophilia product sales for CSL totalled $US558 million ($A767.80 million).

Shares in CSL closed $1.52 higher at $96.17.


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Source: AAP


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