Publication of Clinical Data Demonstrating Low Immunogenicity and Excellent Efficacy of Nuwiq® in Previously Untreated Patie...
August 23 2017 - 2:00AM
Business Wire
Octapharma is delighted to announce the publication of interim
data from the NuProtect study (GENA-05, NCT01712438), the first
clinical data on treatment with Nuwiq® in previously untreated
patients (PUPs), on August 16th 2017 as an Early View article in
the internationally renowned medical journal Haemophilia.
The NuProtect study was initiated in March 2013 to investigate
the immunogenicity, efficacy and safety of Nuwiq® in PUPs with
severe haemophilia A, who are at the greatest risk of developing
inhibitors. The ongoing study has enrolled 110 PUPs of any age or
ethnicity who will receive Nuwiq® for up to 100 exposure days
(EDs), making it one of the largest studies with a single FVIII
product. Patients who previously received any FVIII concentrates or
blood products containing FVIII are excluded from the study.
The recently published article describes interim results for 66
PUPs treated for at least 20 EDs, the time by which most inhibitors
arise. The cumulative incidence (95% confidence interval) was 20.8%
(10.7–31.0) for all inhibitors and 12.8% (4.5–21.2) for high-titre
inhibitors. The study reported excellent efficacy of Nuwiq® in the
prevention of bleeds, with median annual bleeding rates of 0 for
spontaneous bleeds and 2.40 for all bleeds. Nuwiq® was also
efficacious for the treatment of bleeds (92.4% of bleeds were
controlled with one or two infusions) and as surgical prophylaxis
(rated as “excellent” or “good” for 89% of surgical procedures).
These interim results confirm the excellent haemostatic efficacy
reported with Nuwiq® in previously treated patients.
Larisa Belyanskaya, Head of IBU Haematology, said: “We are very
excited by the results of the NuProtect study and pleased that
these interim data are now available as a publication in the
specialist peer-reviewed journal Haemophilia. This is the first
study to report data from PUPs treated with a rFVIII produced in a
human cell line, and the results further differentiate Nuwiq® from
other products on the market.”
Olaf Walter, Board Member at Octapharma, added that “These data
demonstrate low immunogenicity of Nuwiq® in PUPs, the patient group
at the greatest risk of inhibitor development, and confirm the
excellent efficacy observed in previously treated patients. This
publication is a further important step towards Octapharma’s goal
of enabling patients with coagulation disorders to live a normal
life.”
Octapharma would like to thank everyone involved in the study,
in particular the patients and their families, without whom this
research would not be possible.
About the NuProtect (GENA-05) study
The NuProtect clinical study (NCT01712438) is a phase 3,
open-label, interventional clinical study being conducted across 38
centres, to evaluate at least 100 previously untreated patients
(PUPs) with severe haemophilia A of all ages and ethnicities
enrolled for study up to 100 exposure days (EDs) or 5 years
maximum. Patients treated previously with any FVIII
concentrates/blood products containing FVIII are excluded. The
primary objective is to assess the immunogenicity of Nuwiq® by
determining inhibitor activity using the Nijmegen-modified Bethesda
assay at a central laboratory. Final data from the NuProtect study
are expected to become available in 2019. More information on this
trial is available at www.clinicaltrials.gov. An extension study
(GENA-15, NCT01992549) will further evaluate the long-term
immunogenicity, efficacy and safety of Nuwiq® in PUPs.
About Nuwiq®
Nuwiq® is a 4th generation rFVIII protein1, produced in a human
cell line without chemical modification or fusion with any other
protein2. Nuwiq® is cultured without additives of human or animal
origin2, is devoid of antigenic non-human protein epitopes3 and has
a mean half-life of 17.1 hours4,5 and a high affinity for the von
Willebrand coagulation factor6. Nuwiq® treatment has been assessed
in seven completed clinical trials which included 201 PTPs7,8 (190
individuals) with severe haemophilia A, including 59 children9.
Nuwiq® is approved for use in the treatment and prophylaxis of
bleeding across all age groups of PTPs with haemophilia A in the
EU, US, Canada, Australia, Latin America and Russia. Further
worldwide submissions for Nuwiq® are planned.
References:1. FDA Nuwiq® memorandum (STN 1255550), 9 October
2014. 2. Casademunt E, et al. Eur J Haematol 2012; 89: 165-76.3.
Kannicht C, et al. Thromb Res. 2013;131:78-88.4. Nuwiq® European
Public Assessment Report, 22 May 2014.5. NUWIQ® US Prescribing
Information, September 2015.6. Sandberg H, et al. Thromb Res 2012;
130: 808-17.7. Valentino LA, et al. Haemophilia 2014; 20(Suppl. 1):
1-98. Lissitchkov T, et al. Haemophilia 2017; e-pub ahead of print;
doi: 10.1111/hae.132519. Klukowska A, et al. Haemophilia 2016; 22,
232-39.
About Haemophilia A
Haemophilia A is an X-linked hereditary disorder caused by FVIII
deficiency which, if left untreated, leads to haemorrhages in
muscles and joints and consequently to arthropathy and severe
morbidity. FVIII replacement prophylactic treatment reduces the
number of bleeding episodes and the risk of permanent joint damage.
This disorder affects one in every 5,000 to 10,000 men worldwide.
Globally, 75% of haemophilia cases are left undiagnosed or
untreated. The development of neutralising FVIII antibodies (FVIII
inhibitors) against infused FVIII represents the most serious
treatment complication. The cumulative risk of FVIII inhibitor
development is reported to be currently up to 39%.
About Octapharma
Headquartered in Lachen, Switzerland, Octapharma is one of the
largest human protein manufacturers in the world, developing and
producing human proteins from human plasma and human cell lines. As
a family-owned company, Octapharma believes in investing to make a
difference in people’s lives and has been doing so since 1983;
because it’s in our blood.
In 2016, the Group achieved €1.6 billion in revenue, an
operating income of €383 million and invested €249 million to
ensure future prosperity. Octapharma employs more than 7,100 people
worldwide to support the treatment of patients in 113 countries
with products across three therapeutic areas:
- Haematology (coagulation
disorders)
- Immunotherapy (immune disorders)
- Critical care
Octapharma owns six state-of-the-art production facilities in
Austria, France, Germany, Mexico and Sweden.
For more information visit www.octapharma.com
View source
version on businesswire.com: http://www.businesswire.com/news/home/20170822005746/en/
Octapharma AGInternational Business Unit - HaematologyOlaf
WalterOlaf.Walter@octapharma.comorLarisa
BelyanskayaLarisa.Belyanskaya@octapharma.comTel: +41 55 4512121