Novo Nordisk receives positive opinion on recombinant factor XIII from the European regulatory authorities

Novo Nordisk today announced that the Committee for Medicinal Products for Human Use (CHMP) under the European Medicines Agency (EMA) adopted a positive opinion on the Company’s recombinant factor XIII product (rFXIII). The Committee recommended marketing authorisation for rFXIII for once-monthly replacement therapy in patients from the age of six years with congenital factor XIII A-subunit deficiency.

rFXIII is a recombinant factor XIII product, and it is the only recombinant treatment option for congenital factor XIII A-subunit deficiency. Data have shown that rFXIII prevents bleeding and that it is well tolerated.

“The development of rFXIII for a very small patient community with a serious bleeding disorder demonstrates our commitment to develop a broad and innovative pipeline of molecules to fulfil unmet medical needs within haemophilia and rare bleeding disorders,” says Mads Krogsgaard Thomsen, executive vice president and chief science officer at Novo Nordisk.

Novo Nordisk expects to receive the final marketing authorisation from the European Commission within approximately two months. Following the Commission’s approval, Novo Nordisk expects to launch rFXIII in Europe towards end of the year.

About Congenital factor XIII deficiency

Congenital factor XIII deficiency is a rare bleeding disorder with potentially life-threatening consequences, including CNS bleeding, if not treated. Factor XIII is the protein responsible for stabilising the formation of a blood clot. In the absence of factor XIII, a clot will still develop but it will remain unstable. The incidence of factor XIII deficiency is estimated at one in 1-3 million births and affects men and women equally. It is estimated that about 300 patients in Europe and approximately 900 globally are diagnosed with the disease.

About rFXIII

rFXIII (INN name: catridecacog) is the first genetically engineered pro-enzyme dimer that is identical in structure and function to the human FXIII-A subunit. rFXIII has been specifically developed to offer FXIII congenital deficiency patients a new treatment option. rFXIII restores normal clot solubility and increases clot strength and resistance. Because it is genetically engineered, it is not derived from human or mammalian tissue or plasma, and is therefore free from viruses and prions.

rFXIII has also been filed for regulatory review with the FDA (U.S. Food and Drug Administration), Health Canada and SwissMedic.