- The company’s ultra fastacting recombinant factor VIIa is progressing to a phase 3 trial after the successful completion of a phase 2 safety, pharmacokinetics and efficacy trial; the phase 3 trial is currently being designed.聽 A phase 3 trial for the factor XIII drug was recently been completed and showed that, compared to a historic control group of individuals who did not receive prophylactic infusions, treatment with monthly injections with recombinant FXIII significantly decreased the annual number of bleeding episodes requiring treatment.聽 Novo Nordisk expects to file for marketing authorization in the US聽and EU in the first half of 2011.
Details regarding the investigational factor XIII and the ultra fastacting recombinant factor VIIa are included in the company’s quarterly report under “Biopharmaceuticals,” pg 9.聽 http://www.novonordisk.com/include/asp/exe_news_attachment.pdf?sAttachmentGUID=a8c5189e-50be-4393-9e10-9da901dc3c17, and excerpted below.聽
A phase 2 trial evaluating the safety, pharmacokinetics and efficacy of NN1731, an ultra-fastacting recombinant factor VIIa analogue, in treatment of joint bleeds in haemophilia A or B patients with inhibitors has been completed. The trial found that NN1731 has a safe profile for all doses investigated, the ultra-fast-acting profile was confirmed, and NN1731 was efficacious in stopping joint bleeds. Further, all efficacy parameters tested trended favourably for the highest dose of NN1731 compared to NovoSeven®. The control arm confirmed the efficacy and safety profile of NovoSeven® as observed in previous clinical studies. Based on the positive phase 2 trial results, the pivotal phase 3 trial programme for NN1731 is currently being designed.
A pivotal phase 3 trial in factor XIII congenital deficiency, investigating a recombinant FXIII compound, has been finalised. Factor XIII congenital deficiency is a rare bleeding disorder with about 600 diagnosed patients worldwide. This genetic disorder affects both genders and all ethnic backgrounds, and is usually diagnosed at birth. The phase 3 trial enrolled 41 patients for a one-year treatment regimen. The trial proved that recombinant FXIII has a safe profile when administered as prophylactic, monthly replacement therapy to patients with congenital factor XIII deficiency. Compared to a historic control group of individuals who did not receive prophylactic infusions, treatment with monthly injections with recombinant FXIII significantly decreased the annual number of bleeding episodes requiring treatment. Novo Nordisk expects to file for marketing authorisation with the regulatory authorities in the US and EU in the first half of 2011.