by PPN Staff
The FDA has approved Octapharma’s NUWIQ, antihemophilic factor
(recombinant), an IV therapy for adults and children living with
hemophilia A. The approval includes on-demand treatment and control
of bleeding episodes, routine prophylaxis to reduce the frequency of
bleeding episodes and perioperative management of bleeding.
NUWIQ is the first B-domain–deleted recombinant factor VIII (FVIII) derived from a human cell line, not chemically modified or fused with another protein, designed for the treatment of patients with hemophilia A, congenital FVIII deficiency.
Up to 16,000 individuals in the United
States have hemophilia A. Although there are already therapies for
hemophilia A, significant challenges remain, including development of
inhibitors and the need for multiple infusions on a prophylactic
basis, the company said.
The initial global clinical study
program for NUWIQ commenced with a pharmacokinetic (PK) evaluation in
an open-label, multicenter clinical trial of 22 previously treated
patients (PTPs). These patients consisted of 20 adults and two
adolescents. In this study, NUWIQ demonstrated a mean half-life of
17.1 hours using a one-stage clotting assay in adults. NUWIQ was also
evaluated in children using a one-stage clotting assay with a mean
half-life of 11.9 hours for ages 2 to 5 years and a mean half-life of
13.1 hours for ages 6 to 12 years. These PK results for mean
half-life were longer than earlier generations of recombinant FVIII
products currently available in the United States.
The second set of global clinical
studies for NUWIQ also evaluated overall efficacy and tolerability in
three prospective, open-label clinical studies in PTPs with severe
hemophilia A. From all clinical studies, 135 patients were treated
with NUWIQ, including 74 adults, three adolescents between ages 12
and 17 years and 58 pediatric patients between ages 2 and 11 years.
All these patients were treated with a total of 16,134 infusions over
15,950 exposure days using NUWIQ.
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 with 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 all of 30
(100%) bleeds in adults and 89 of 108 bleeds (82%) in children. For
on-demand treatment with NUWIQ in 20 adults and two adolescents,
efficacy for the treatment of bleeds was excellent or good in 931 of
986 bleeds (94%). Overall efficacy in surgical prophylaxis was rated
excellent or good in 32 of 33 procedures (97%) using NUWIQ.
In all clinical studies, NUWIQ had a
total of seven reported adverse events. Each of these adverse events
occurred one time with a rate of 0.7% across all 135 patients. These
events were parathesia, headache, injection site inflammation,
injection site pain, back pain, vertigo and dry mouth.
Octapharma USA will offer hemophilia A
patients educational and support services in connection with the
introduction of NUWIQ, which should be available by early 2016.
NUWIQ is not indicated for the
treatment of vonWillebrand disease.
“We are pleased that the treatment
options for adults and children with hemophilia A continue to
advance with ever more innovative therapies being approved for the
U.S.,” said Val Bias, CEO of the National Hemophilia Foundation.
“The continued commitment to develop life-enhancing products for
the bleeding-disorders community is absolutely vital. Empowering
patients and providers with treatment options, as well as education
and support programs, is extremely important to people living with
hemophilia A.”
—From company press
materials
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