Octapharma announced the U.S. Food and Drug Administration (FDA) has approved Nuwiq (recombinant Factor VIII) for adults and children with Hemophilia A.
Nuwiq is indicated for:
on-demand treatment and control of bleeding episodes
routine prophylaxis to reduce the frequency of bleeding episodes
perioperative management of bleeding.
Octapharma USA President Flemming Nielsen said in a press release, “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. Octapharma is dedicated to providing life enhancing and saving therapies for Hemophilia A and looks forward to bringing Nuwiq to the U.S. marketplace.”
Nuwiq was approved in Europe in August 2014 and is currently approved in many countries, including the United Kingdom, Australia, Canada, Germany, Italy, Sweden and Argentina.
Efficacy of Nuwiq
Studies indicate Nuwiq has a mean half-life of 17.1 hours in adults, 11.9 hours in children ages 2 to 5; and a 13.1 hours in children ages 6 to 12.
The efficacy and tolerability of Nuwiq was demonstrated in 3 prospective, open-label clinical studies in patients with severe Hemophilia A.
In a study of 32 adults, overall prophylactic efficacy of Nuwiq for spontaneous bleeds was rated as excellent or good in 92% of patients. In a study of 59 children, prophylactic efficacy for spontaneous bleeds was rated as excellent or good in 97% of patients.
The mean annualized bleeding rates (ABR) for spontaneous bleeds during prophylaxis were approximately 1.5 in children and 1.2 in adults.
For Hemophilia A patients receiving Nuwiq prophylaxis compared to on-demand treatment, the ABR was reduced 96% for adults and 93% for children.
Treatment of breakthrough bleeds during Nuwiq prophylaxis was rated as excellent or good in 30 of 30 (100%) bleeds in adults and for 89 of 108 (82%) bleeds in children.
For on-demand treatment with Nuwiq in 20 adults and 2 adolescents, efficacy for the treatment of bleeds was 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.
Safety of Nuwiq
In all clinical studies, 7 adverse events were reported. Each of these side effects occurred 1 time with a rate of 0.7% across all 135 patients. Adverse events were parathesia, headache, injection site inflammation, injection site pain, back pain, vertigo, and dry mouth.
When will Nuwiq be available?
Octapharma USA plans to have Nuwiq available in the United States by early 2016.
What is Hemophilia A?
Hemophilia A is a rare, chronic, genetic disorder that results in impaired clotting mechanisms due to missing or reduced levels of factor VIII. People with hemophilia A experience recurrent and extended bleeding episodes that cause pain and irreversible joint damage. Some of these bleeding episodes can be life-threatening. There are an estimated 142,000 people worldwide with hemophilia A. Many orphan drugs are approved to help in the management of patients with hemophilia A, including Eloctate
FDA Approves Octapharma’s NUWIQ® for the Treatment of Adults and Children with Hemophilia A [news release]. Hoboken, NJ: Octapharma USA; Septeber 15, 2015. http://www.octapharma.com/en/about/newsroom/press-releases/news-single-view.html?tx_ttnews[tt_news]=676&cHash=f43f9dcd137c0d309fadc28b1749e315