Octapharma AG: Final data from the NuProtect study published on the immunogenicity of Nuwiq(R) in previously untreated patients with severe haemophilia A

PR88178

LACHEN, Switzerland, Feb. 23, 2021 /PRNewswire=KYODO JBN/ --

Octapharma announced today that the final results from the NuProtect study on

the immunogenicity of Nuwiq(R) in previously untreated patients (PUPs) with

severe haemophilia A have been published in the leading medical journal

Thrombosis and Haemostasis (Liesner RJ et al. "Simoctocog Alfa (Nuwiq(R)) in

Previously Untreated Patients with Severe Haemophilia A: Final Results of the

NuProtect Study"

https://www.thieme-connect.com/products/ejournals/abstract/10.1055/s-0040-1722623).

The NuProtect study (NCT01712438; EudraCT 2012-002554-23) was a prospective,

multinational, open-label, non-controlled phase III study initiated in March

2013 to assess the immunogenicity, efficacy and safety of Nuwiq(R). The study

recruited patients of any age and ethnicity at 38 sites in 17 countries and

followed patients for 100 exposure days or up to 5 years. With enrolment of 110

patients, the NuProtect study was the largest clinical study to investigate a

single product in true PUPs.

Of 105 evaluable PUPs with severe haemophilia A who received Nuwiq(R) for the

prevention and treatment of bleeding:

- 16.2% (17/105) of patients developed high-titre inhibitors

- 10.5% (11/105) of patients developed low-titre inhibitors, five of which were

transient

- 26.7% (28/105) of patients developed any inhibitor

"The NuProtect study showed that there was a low risk of inhibitor development

in PUPs starting treatment with Nuwiq(R)," commented Dr Ri Liesner,

coordinating investigator of the NuProtect study and clinician at Great Ormond

Street Hospital for Children in London, UK. "These data indicate Nuwiq(R) may

be an attractive option for people newly diagnosed with severe haemophilia A,

who are young children and represent a vulnerable patient subset."

The development of inhibitors to replacement factor VIII (FVIII) is a concern

for doctors, as well as for patients and their families, particularly when

first starting treatment. Inhibitors render FVIII therapy ineffective and limit

treatment options.

These data complement the wealth of clinical experience in patients with

haemophilia A receiving Nuwiq(R) for the treatment and prevention of bleeding.

"Nuwiq(R) was developed in a human cell line with the aim of minimising

inhibitor risk in PUPs," said Larisa Belyanskaya, Head of Octapharma's IBU

Haematology. "We are pleased to be able to share these positive data, which

reflect this aim and which we hope will contribute to addressing this key

treatment challenge."

"We know that haemophilia A patients face a lifetime of treatment decisions,"

commented Olaf Walter, Board Member at Octapharma. "These data highlight the

potential of Nuwiq(R) to reassure PUPs and their families as they start their

treatment. This publication brings us closer to Octapharma's goal of enabling

all patients to lead healthy lives."

Earlier this year the FDA approved the inclusion of the immunogenicity data

from the NuProtect study in the Nuwiq(R) Prescribing Information. Further

publications on other analyses from the NuProtect study are planned.

Professor Anthony Chan, a co-author of the publication and Professor of

Paediatrics at McMaster University, Canada, said: "Congratulations and thanks

to Octapharma for developing Nuwiq, sponsoring the trial and providing a very

good therapy for patients with haemophilia A."

Octapharma would also like to extend thanks to all participating centres, as

well as patients and their caregivers, for their contribution to the study.

About Nuwiq(R)

Nuwiq(R) (simoctocog alfa) is a 4th generation recombinant factor VIII (rFVIII)

protein, produced in a human cell line without chemical modification or fusion

with any other protein1. It is cultured without additives of human or animal

origin, is devoid of antigenic non-human protein epitopes and has a high

affinity for von Willebrand factor1. Nuwiq(R) treatment has been assessed in

seven completed clinical trials which included 201 previously treated patients

(PTPs; 190 individuals) with severe haemophilia A, including 59 children1.

Nuwiq(R) is available in 250 IU, 500 IU, 1000 IU, 2000 IU, 2500 IU, 3000 IU and

4000 IU presentations2. Nuwiq(R) is approved for use in the treatment and

prophylaxis of bleeding in patients with haemophilia A (congenital FVIII

deficiency) across all age groups2.

1.  Lissitchkov T et al. Ther Adv Hematol 2019; 10:2040620719858471.

2.  Nuwiq (R) Summary of Product Characteristics.

About Haemophilia A

Haemophilia A is an X-linked hereditary bleeding disorder caused by a

deficiency of factor VIII (FVIII) which, if left untreated, may lead to

haemorrhages in muscles and joints and consequently to arthropathy and severe

morbidity. The disorder affects around one in every 10,000 males worldwide.

Prophylaxis with replacement FVIII therapy reduces the number of bleeding

episodes and the risk of permanent joint damage.  

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.

Octapharma employs more than 9,000 people worldwide to support the treatment of

patients in 118 countries with products across three therapeutic areas:

Haematology, Immunotherapy, and Critical Care.

Octapharma has seven R&D sites and six state-of-the-art manufacturing

facilities in Austria, France, Germany, Mexico and Sweden, and operates more

than 160 plasma donation centres across Europe and the USA.

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SOURCE: Octapharma AG