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Novo Nordisk to build on heritage in rare blood disorders

New data will be released at the International Society on Thrombosis and Haemostasis Congress.

Novo Nordisk is advancing its ambition to drive change in rare diseases with new data due to be revealed at the International Society on Thrombosis and Haemostasis 2022 Congress, which takes place in London in July.

A total of three molecules across fifteen abstracts will be presented at the congress, including data for the monoclonal antibody, concizumab, and next-generation FVIIIa mimetic, Mim8. The presentations will further reinforce Novo Nordisk’s expansive haemophilia portfolio.

In a late-breaking oral presentation, the efficacy and safety results from the primary analysis of the phase 3 study of concizumab, which involves people living with haemophilia A or B with inhibitors, will be analysed and discussed.

Based on the outcomes of phase 2 studies, the treatment experience of once-daily concizumab prophylaxis versus on-demand therapies – as assessed by Haemophilia Treatment Experience Measure (Hemo-TEM) scores – will also be presented.

Meanwhile, data from the phase 1/2 study of Mim8 – a once-weekly or monthly treatment for people living with haemophilia A – will help set the stage for the future of this investigational molecule.

Patients managing haemophilia B with inhibitors currently have limited treatment options, while the use of concizumab and Mim8 in haemophilia is investigational and not currently approved by regulatory authorities.

In addition, data from the paradigm study in previously untreated children with haemophilia B under six years of age, treated with once-weekly Refixia prophylaxis for up to six years, will be shared at the congress.

The use of Refixia in children below the age of 12 years old living with haemophilia B is also not approved by regulatory authorities.

“Our continued drive for innovation in rare blood disorders, through next-generation medicines and novel applications for our existing medicines, reflects our commitment to transforming the lives of people living with a rare blood disorder,” explained Ludovic Helfgott, executive vice president and head of Novo Nordisk Rare Disease. “We are proud of our heritage in haemophilia and are committed to addressing the changing, complex and individual needs of people living with this rare disease.”