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Bayer files haemophilia drug in EU

pharmafile | December 5, 2014 | News story | Sales and Marketing BAY 81-8973, Bayer, Biogen IDEC, EMA, elocta, haemophilia a, kogenate 

Bayer has submitted a marketing authorisation application to the EMA for its haemophilia A therapy BAY 81-8973.

The German firm’s recombinant factor VIII (RfvIII) compound has demonstrated positive results when used with typical dosages for prophylaxis, two or three times a week.

The submission is based on the encouraging upshots from LEOPOLD II, a Phase III trial involving 205 children, adolescents and adults.

“Clinical data from the trials show that BAY 81-8973 provides bleeding control in people with severe haemophilia when infused either twice per week or three times per week – with typical dosages,“ says lead investigator Johannes Oldenburg, head of the University of Bonn’s institute of experimental haematology and transfusion medicine.

Results show a 93.3% reduction in median annualised bleeding rate in the twice-per-week prophylaxis arm, versus on-demand and a 96.7% reduction in the three-times-per-week arm versus on-demand.

Haemophilia A is characterised by prolonged or spontaneous bleeding, especially into the muscles, joints or internal organs. It is the most common type of haemophilia and is caused by deficient or defective blood coagulation proteins, known as factor VIII.

The 2012 World Federation of Haemophilia global survey estimated that around 142,000 people worldwide live with haemophilia A.

“Bayer has long had a strong commitment to the haemophilia community, and the submission of BAY 81-8973 is an important step forward in our efforts to improve treatment options for people with haemophilia,” says Dr Joerg Moeller, member of the Bayer healthcare executive committee and head of global development.

Elocta: one step ahead

Already approved in Canada, Australia and the US, Biogen Idec and Sobi’s own haemophilia A therapy Elocta (rFVIIIFc) is a stride forward in the race for EU approval – as their drug has already been validated for EMA submission.

Douglas Williams, executive vice president of R&D at Biogen Idec, says: “The validation of Elocta’s application by the EMA is an important step toward bringing this innovative treatment to people with haemophilia A in Europe.”

The drug is being evaluated for use in children and adults, both of which were studied under the Phase III A-LONG trials. The studies only looked at male participants, however.

Birgitte Volck, senior vice president of development and chief medical officer of Sobi, concludes: “We’re working collaboratively to deliver innovative medicines that have the potential to change the way haemophilia A is treated.”

Tom Robinson

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