FDA Approves Multiple Hemophilia A Indications for Octapharma’s NUWIQ®
The US Food and Drug Administration (FDA) recently approved Octapharma’s new therapy NUWIQ®, for the treatment of bleeding in children and adults with hemophilia A. The product is indicated for on-demand treatment and control of bleeding episodes; routine prophylaxis to reduce the frequency of bleeding episodes; and perioperative management of bleeding. According to Octapharma, NUWIQ® is the first B-domain deleted recombinant factor VIII derived from a human cell-line. The therapy is cultured without using additives of human or animal origin, and is not chemically modified or fused with another protein.
The FDA’s approval of NUWIQ® was granted in light of several successful global studies demonstrating the product’s pharmacokinetics (PK), efficacy and tolerability. A multi-center PK clinical trial of previously treated patients (PTPs) with hemophilia A showed a mean half-life of 17.1 hours in adults, 11.9 hours for children ages 2 to 5, and 13.1 hours for children ages 6 to 12.
A subsequent series of clinical studies measured overall efficacy and tolerability of NUWIQ® in 135 adult and pediatric PTPs with severe hemophilia A. Overall, these patients were treated with a total of 16,134 infusions of the product over 15,950 exposure days. One study rated NUWIQ®’s prophylactic efficacy in 32 adults and found that the product’s capacity to control spontaneous bleeds was “excellent or good” in 92% of the participants. In another study, prophylactic efficacy for spontaneous bleeds was rated as excellent or good in 97% of the 59 pediatric patients enrolled.
The mean annualized bleeding rates (ABR) for spontaneous bleeds during these prophylaxis studies were approximately 1.5 in children and 1.2 in adults. In addition, treatment of breakthrough bleeds was rated as excellent or good in 30 of 30 (100%) bleeds in adults and for 89 of 108 (82%) bleeds in children. On-demand efficacy of NUWIQ® to treat bleeds in 20 adults and 2 adolescents was rated excellent or good in 931 of 986 (94%) bleeds. Overall efficacy in surgical prophylaxis was rated excellent or good in 32 of 33 (97%) procedures using NUWIQ®. Lastly, none of the patients in these studies developed inhibitors.
“Early development strategies were integral in the development of NUWIQ® and these initial goals have been realized with the FDA approval,” said Octapharma USA President Flemming Nielsen. “NUWIQ® has demonstrated safety and efficacy in global clinical trials and has the potential to positively impact patients’ quality of life in the years ahead.”
Source: Reuters, September 15, 2015