‘World Hemophilia Day’ marked by release of data

‘World Hemophilia Day’ occurred on 17 April and the day was marked by the release of data from two companies involved in treating the condition. Shire took the opportunity to reveal that a ‘bleeding episode’ occurs every three to fifteen seconds worldwide. Shire’s report noted that this is more than three times the previous estimate for the Global Annual Bleed Rate (GABR).

Shire reported these findings alongside data from the World Federation of Hemophilia that only 25% of those suffering from haemophilia are diagnosed with the condition and only 8% receive treatment to manage their condition.

"As the global leader in haematology, we are committed to efforts, such as estimating the GABR, that improve our understanding of the global landscape for haemophilia and the true experience of those living with this debilitating bleeding disorder," said Leonard Valentino, Global Head of Haematology Medical Affairs, Shire.

Shire became one of the biggest drugmakers in the competitive haemophilia market after its merger with Baxalta. It suffered poor sales in the area in the third quarter of last year but recovered recently, with results posted in February showing improvement.

The market is estimated to be worth $11 billion per year and there are five primary companies involved in producing treatments: Shire, Bayer, CSL, Novo Nordisk and Bioveratic (a spin-off of Biogen).

However, there may be another company poised to shake up the market. Roche used World Hemophilia Day to post positive interim results from a Phase 3 study of its haemophilia drug, ACE910 (emicizumab prophylaxis). The study looked into the treating children with the illness, who find it particularly difficult to manage their condition.

"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.

The drug is potential game-changer for treatment and is being closely watched by competitors. ACE109’s form of action is to inhibit clotting protein factor VIII, which could mean managing the condition in a different, and more convenient, way to traditional treatments.