[August 06, 2014] |
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European Commission publishes approval of Octapharma's human cell line recombinant FVIII (Nuwiq®) across all age groups in haemophilia A
LACHEN, Switzerland --(Business Wire)--
The European Commission has published approval of Octapharma's Nuwiq®
(simoctocog alfa) for the treatment and prophylaxis of bleeding in all
age groups with haemophilia A (congenital factor VIII [FVIII]
deficiency). Nuwiq® is the newest generation of FVIII
replacement, produced in a human cell line cultured without additives of
human or animal origin. Nuwiq® is devoid of antigenic
non-human protein epitopes and has a high affinity for the von
Willebrand coagulation factor, both of which are potentially important
properties in reducing FVIII inhibitor formation. The development of
Nuwiq® aimed to address the challenges of inhibitor formation
as well as the frequent infusions required for prophylaxis of bleeding.
"Nuwiq® represents a potential leap forward in dealing with
the greatest clinical challenges in haemophilia A - FVIII inhibitors and
the frequent infusions needed for bleeding prophylaxis. In clinical
trials, Nuwiq® demonstrated outstanding efficacy in
preventing and treating bleeding in patients previously on FVIII (PTPs).
No inhibitors in clinical trials in PTPs have been seen", says Olaf
Walter, MD, PhD, MBA, Senior Vice President of International Business
Units.
The benefit of Nuwiq® in terms of prevention and treatment of
bleedings and haemostatic efficacy for surgical procedures was studied
in three pivotal trials. In a study of 32 adults (GENA-08), overall
prophylactic efficacy of Nuwiq® forspontaneous bleeds was
rated as excellent in all (100%) patients. In a study of 59 children
(GENA-03), prophylactic efficacy for spontaneous bleeds was rated as
excellent or good in 96.6% of patients. Mean annualised bleeding rates
for spontaneous bleeds during prophylaxis were approximately 1.5 in
children and 1.2 in adults. Treatment of breakthrough bleeds during
prophylaxis was rated as excellent or good for 28 of 28 (100%) bleeds in
adults and for 89 of 108 (82.4%) bleeds in children. In on-demand
treatment with Nuwiq® in 20 adults and 2 adolescents
(GENA-01), efficacy for the treatment of bleeds was excellent or good in
931 of 986 (94.4%) bleeds. Overall efficacy across the GENA programme in
surgical prophylaxis was rated excellent or good in 32 of 33 (97%)
procedures.
The immunogenicity of Nuwiq® was evaluated in 135 PTPs (74
adults and 61 children). None of the patients developed inhibitors.
The next steps in Nuwiq®'s clinical programme include
continued investigation of previously untreated patients (PUPs), with a
target enrolment of 100 patients in the ongoing NuProtect study. PUPs
are typically characterised by a higher risk of inhibitors. Octapharma
is also committed to the ongoing NuPreviq study in PTPs, which is
designed to test a robust programme of personalised prophylaxis, with a
potential reduction in the number of FVIII infusions.
Regulatory data for Nuwiq® have been submitted to authorities
in Canada, the US, and Australia, with further worldwide submissions
planned.
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 AG
Headquartered in Lachen, Switzerland, Octapharma AG is one of the
largest human protein products manufacturers in the world and has been
committed to patient care and medical innovation for more than 30 years.
Its core business is the development, production and sale of human
proteins from human plasma and human cell-lines. Patients in more than
100 countries are treated with products in the following therapeutic
areas:
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Haematology (coagulation disorders)
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Immunotherapy (immune disorders)
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Critical Care
Octapharma owns five state-of-the-art production facilities in Austria,
France, Germany, Sweden and Mexico.
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