Data Supports Potential for Personalized Hemophilia A Therapy with Nuwiq, WFH Presentations Show

Patricia Inácio, PhD avatar

by Patricia Inácio, PhD |

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Since every hemophilia A patient responds differently to therapies, researchers are working to develop personalized preventive (prophylaxis) strategies with Nuwiq (antihemophilic factor [recombinant].)

Octapharma, the treatment’s manufacturer, presented data showing that an individual’s pharmacokinetic (PK) profile (how the therapy works once inside the body) can be used to possibly develop a customized treatment.

The results were shared in the symposium “Going further to meet clinical needs: New data with Nuwiq (simoctocog alfa; human-cl rFVIII) from clinical trials and real-world experience, that took place at the World Federation of Hemophilia (WFH) 2018 World Congress  in Glasgow.

Nuwiq is a fourth-generation genetically engineered version of the blood-clotting factor VIII, developed to safely and effectively control bleeding in people with hemophilia A.

In the first presentation, John Pasi with The Royal London Hospital presented results of a Phase 3b multicenter study (NCT01863758), which followed 66 previously treated adults with severe hemophilia A to assess the benefit and safety of a personalized prophylaxis with Nuwiq. The program used each individual’s PK profile to optimize treatment.

For 57% of the patients, the personalization led to reducing treatment frequency to twice weekly, without compromising the therapy’s benefit. In fact, during personalized prophylaxis, 83% of patients were bleed free.

In a second presentation, Stacy Croteau with Boston Children’s Hospital shared an analysis for predicting optimal treatment for hemophilia A patients using the Web Accessible Population Pharmacokinetics Program or WAPPS.

Based on the PK profile of 114 patients, researchers hope it will also help in developing a Nuwiq-personalized hemophilia care.

Ellis Neufeld with St. Jude Children’s Research Hospital in Memphis shared interim data from the Phase 3 NuProtect study (NCT01992549) that is investigating the effects of Nuwiq in 100 previously untreated patients for 100 exposure days, or 5 years maximum.

Among 66 patients exposed for more than 20 days to Nuwiq, 12.8% developed high-titre (concentration) inhibitors, antibodies that prevent Factor VIII treatments from working to control bleeding.

For patients with high-risk mutations in the factor VIII gene, the incidence of inhibitors was of 26.7%, while patients with low-risk mutations didn’t develop inhibitors.

Dan Hart, also with The Royal London Hospital, presented a new approach using gene expression (activity) analysis that might predict a person’s risk of developing inhibitors.

Ri Liesner with Great Ormond Street Hospital for Children in London presented real-world data of Nuwiq as an immune tolerance induction (ITI) therapy. Data from five patients showed that three are inhibitor free, and levels are steadily declining in the other two.

“We at Octapharma are delighted with the excellent data presented at WFH 2018, which build on the growing wealth of positive experience with Nuwiq. The broad range of data demonstrate the expanding value of Nuwiq in clinical practice and highlight Octapharma’s commitment to ‘going further’ to address the developing demands of haemophilia A patients,” Larisa Belyanskaya, head of Octapharma’s IBU Haematology, said in a press release.

Nuwiq, an intravenous therapy, was approved by the U.S. Food and Drug Administration in 2015 to treat adults and children with hemophilia A.