LACHEN, Switzerland--(BUSINESS WIRE)--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 haemophilia community”.
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® are 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%.
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 therapeutic areas:
• 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.