Octapharma announced today that the European Medicines Agency (EMA) has
approved an extension of marketing authorisation for its human cell
line-derived recombinant factor VIII (rFVIII) product, Nuwiq®.
New single dose vial strengths of 2500, 3000 and 4000 International
Units (IU) will be available in Europe, in addition to the current
strengths of 250, 500, 1000 and 2000 IU.
The new Nuwiq® vial strengths have been available in the US
since September last year, following approval by the FDA. Nuwiq®
is indicated in adults and children of all ages for on-demand treatment
and prophylaxis, including during surgery, to prevent and control
bleeding episodes in patients with haemophilia A.
This expanded array of vial strengths has the potential to increase
treatment options for patients with haemophilia A by improving dosing
flexibility. Patients who previously required more than one vial per
infusion may be able to reduce the number of vials needed. Nuwiq®
is the only rFVIII available in an extended range of vial sizes that are
reconstituted in a volume of 2.5 mL.
The new vial sizes could be particularly beneficial to patients treated
with Nuwiq® based on a pharmacokinetic (PK)-guided
personalised prophylaxis approach. The NuPreviq Approach uses each
individual’s own PK profile to tailor the treatment plan to the
individual patient. In the NuPreviq study, the use of this approach
enabled over half (57%) of patients to reduce Nuwiq® dosing
to twice weekly or less, whilst maintaining effective bleed protection
(median annualised bleeding rate for all bleeds: 0 )1. During
personalised prophylaxis with Nuwiq®, 83% of patients were
spontaneous bleed free. A second approach to individualising dosing uses
PK analysis of a group of people to predict the optimal treatment for an
individual; a Nuwiq®-specific predictive model is available
as part of the Web Accessible Population Pharmacokinetics Service –
Hemophillia (WAPPS-Hemo). The increase in vial options for Nuwiq®
could enable physicians to more closely align to a specific,
individualised dosing regimen by combining vial strengths, allowing more
flexibility in dosing, and supporting the optimisation and
personalisation of haemophilia care.
Larisa Belyanskaya, Head of Octapharma Haematology IBU, said “the
increase in vial strength options will benefit patients and physicians
by providing further treatment flexibility and convenience with Nuwiq®.
As personalised prophylaxis becomes a major part of haemophilia
treatment, this flexibility will simplify dosing to facilitate the
optimisation of treatment for every individual patient”. Olaf Walter,
Board Member of Octapharma, added that “this change represents another
mark of Octapharma’s ongoing commitment to improving the lives of the
Nuwiq® is a 4th generation rFVIII protein2,
produced in a human cell line without chemical modification or fusion
with any other protein3. Nuwiq® is cultured
without additives of human or animal origin3, is devoid of
antigenic non-human protein epitopes4 and a high affinity for
the von Willebrand coagulation factor5. Nuwiq®
treatment has been assessed in seven completed clinical trials which
included 201 PTPs6,7 (190 individuals) with severe
haemophilia A, including 59 children8. 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®
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%.
The vision of Octapharma is “Our passion drives us to provide new health
solutions advancing human life”. 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. Our company values are Ownership,
Integrity, Leadership, Sustainability and Entrepreneurship.
In 2017, the Group achieved €1.72 billion in revenue, an operating
income of €349 million and invested €287 million to ensure future
prosperity. Octapharma employs around 7,700 people worldwide to support
the treatment of patients in 113 countries with products across three
• Haematology (coagulation disorders)
• Immunotherapy (immune disorders)
• Critical care
Octapharma has seven R&D sites and six state-of-the-art manufacturing
facilities in Austria, France, Germany, Mexico and Sweden.
For more information visit www.octapharma.com
1. Lissitchkov T, et al. Haemophilia 2017;23:697-704.
2. Lieuw K. J Blood Med 2017; 8: 67–73.
3. Casademunt E, et al. Eur J Haematol 2012; 89: 165-76.
4. Kannicht C, et al. Thromb Res. 2013; 131: 78-88.
5. Sandberg H, et al. Thromb Res 2012; 130: 808-17.
6. Valentino LA, et al. Haemophilia 2014; 20(Suppl. 1): 1-9.
7. Lissitchkov T, et al. Haemophilia 2017; 23: 697–704.
8. Klukowska A, et al. Haemophilia 2016; 22, 232-39.
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