Swiss company's hemophilia drug shows positive results with children

Managing the disease is especially challenging for children and their caregivers as the bleeding is difficult to control and current treatments require frequent infusions, the company said

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Reuters
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Swiss company's hemophilia drug shows positive results with children
The logo of Swiss pharmaceutical company Roche is seen outside their headquarters in Basel, January 30, 2014. To match special report USA-FDA/CASES REUTERS/Ruben Sprich/File Photo

Swiss drug manufacturer Roche, which is carrying out tests with emicizumab – its drug to treat haemophilia A, revealed on Monday that the third trial has been successful in with children with inhibitors to clotting protein factor VIII.

Roche Holding AG hopes to win a slice of the $11-billion-a-year haemophilia drug market with the new drug, which is designed to compete with more traditional treatments from peers. The under-test medication is being closely watched because it could change the way the disease is treated.

Haemophilia patients, whose blood does not clot properly, need life-saving infusions of clotting factors, but the development of inhibitors in many of those being treated can interfere with efforts to control their bleeding.

"At this interim analysis after a median of 12 weeks of treatment, emicizumab prophylaxis showed a clinically meaningful reduction in the number of bleeds over time," Roche said in a statement.

Roche said managing haemophilia A with inhibitors to factor VIII was especially challenging for children and their caregivers because the bleeding was difficult to control and current treatments required frequent intravenous infusions. Earlier, in December, it published encouraging data on the drug from a phase III study with adults.