LACHEN, Switzerland-Thursday 12 December 2019 [ AETOS Wire ]
(BUSINESS WIRE) -- Octapharma has announced today that the final results from the NuProtect study, the largest prospective study of a single factor VIII (FVIII) product in true PUPs with severe haemophilia A, were presented today in an oral presentation at the American Society of Hematology (ASH) 2019 Annual Meeting in Orlando, Florida.
The NuProtect study was a prospective, multinational, open-label, non-controlled phase III study to assess the immunogenicity, efficacy and safety of Nuwiq® (simoctocog alfa), a human cell line-derived recombinant FVIII, in PUPs with severe haemophilia A. Dr Ri Liesner from Great Ormond Street Hospital for Children, London, UK, presented for the first time the final data on the immunogenicity, efficacy and safety of Nuwiq® (simoctocog alfa) in PUPs followed for up to 100 exposure days.
Dr Liesner, the Principal Investigator of the study, commented that “the NuProtect study has made a valuable contribution to our understanding of factors affecting inhibitor development in haemophilia A and should help to inform treatment decisions in PUPs”.
When treating PUPs with severe haemophilia A, the development of neutralising antibodies against FVIII is the most significant concern. In 105 patients in the NuProtect study, the cumulative incidence of high-titre inhibitors was 17.6% and the cumulative incidence of all inhibitors was 27.9%. Dr Liesner discussed these results in the context of data from the SIPPET study, in which the cumulative incidence of high-titre inhibitors was 28.4% in patients treated with recombinant FVIII products derived from hamster cell lines, and 18.6% in those treated with plasma-derived FVIII products.
Interestingly, an analysis of the underlying F8 gene mutation demonstrated that no patients with non-null mutations developed inhibitors to Nuwiq® in the NuProtect study.
“The data suggest that Nuwiq® has the potential for low immunogenicity in PUPs, with a profile more similar to plasma-derived FVIII products than to recombinant FVIII products derived from hamster cells”, commented Dr Liesner. “This relatively large clinical data set provides support for the use of Nuwiq® in PUPs”.
The data also demonstrated the efficacy and safety profiles of Nuwiq® for the prevention and treatment of bleeding in PUPs. Efficacy in the prevention of spontaneous bleeding events was rated as “excellent” in all 50 patients receiving continuous prophylaxis with Nuwiq®. On-demand treatment was rated as “excellent” or “good” for 93% of bleeding episodes, and efficacy as surgical prophylaxis was rated as “excellent” or “good” for 95% of 19 rated surgeries. Nuwiq® was well-tolerated, with only one possibly treatment-related serious adverse event (a mild rash requiring hospitalisation) reported by investigators, which resolved and treatment was continued.
Dr Larisa Belyanskaya, Head of IBU Haematology at Octapharma, said that “we are proud to be able to communicate such exciting data for Nuwiq® in this challenging patient group. This landmark study adds to the broad clinical experience with Nuwiq® and represents an important step in demonstrating the value of Nuwiq® for patients with haemophilia A at all clinical stages, from the very first treatment to long-term protection”.
Olaf Walter, Board Member at Octapharma, added that “We believe that the immunogenicity profile of Nuwiq® reflects its human cell line origin, and more broadly the foundations of Octapharma’s approach to using human proteins to improve the lives of patients. These data demonstrate the value of our strategy and show Octapharma’s ongoing commitment to the bleeding disorders community”.
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 Nuwiq®
Nuwiq® (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® 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. Treatment of previously untreated patients (PUPs) with Nuwiq® was assessed in the NuProtect study. In an interim analysis of 66 patients, the cumulative incidence of high-titre inhibitors was 12.8%, and the cumulative incidence of all inhibitors was 20.8%2. Nuwiq® is available in 250 IU, 500 IU, 1000 IU, 2000 IU, 2500 IU, 3000 IU and 4000 IU presentations3. Nuwiq® is approved in 59 countries for use in the treatment and prophylaxis of bleeding in patients with haemophilia A (congenital FVIII deficiency) across all age groups3.
About the NuProtect study
The NuProtect study (NCT01712438) was a prospective, multicentre, multinational, open-label, non-controlled phase III study to assess the immunogenicity, efficacy and safety of Nuwiq® in patients with severe haemophilia A and without any previous exposure to FVIII concentrates or blood products containing FVIII. Patients were treated with Nuwiq® for 100 exposure days or up to 5 years. The study was conducted at 38 sites in 17 countries. A total of 110 patients were enrolled. Of these, 108 patients were treated with Nuwiq®.
About Octapharma
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 2018, the Group achieved €1.8 billion in revenue, an operating income of €346 million and invested €240 million into R&D and in capital expenditures in order to ensure future prosperity. Octapharma employs 8,314 people worldwide to support the treatment of patients in 115 countries with products across three therapeutic areas:
Haematology (coagulation disorders)
Immunotherapy (immune disorders)
Critical care (bleeding management and functional volume replacement)
Octapharma has seven R&D sites and six state-of-the-art manufacturing facilities in Austria, France, Germany, Mexico and Sweden.
1. Lissitchkov T et al. Ther Adv Hematol 2019; doi: 10.1177/2040620719858471.
2. Liesner R et al. Haemophilia 2018; 24:211-20.
3. Nuwiq® Summary of Product Characteristics.
View source version on businesswire.com: https://www.businesswire.com/news/home/20191210005421/en/
Contacts
Octapharma AG:
Octapharma AG International Business Unit - Haematology
Olaf Walter
Olaf.Walter@octapharma.com
Larisa Belyanskaya
Larisa.Belyanskaya@octapharma.com
Tel: +41 55 4512121
Ivana Spotakova
Communications Manager
ivana.spotakova@octapharma.com
Tel.: +41793474607
Permalink : https://www.aetoswire.com/news/final-results-from-the-nuprotect-study-demonstrating-efficacy-of-nuwiqreg-simoctocog-alfa-in-previously-untreated-patients-pups-presented-at-ash-2019/en
(BUSINESS WIRE) -- Octapharma has announced today that the final results from the NuProtect study, the largest prospective study of a single factor VIII (FVIII) product in true PUPs with severe haemophilia A, were presented today in an oral presentation at the American Society of Hematology (ASH) 2019 Annual Meeting in Orlando, Florida.
The NuProtect study was a prospective, multinational, open-label, non-controlled phase III study to assess the immunogenicity, efficacy and safety of Nuwiq® (simoctocog alfa), a human cell line-derived recombinant FVIII, in PUPs with severe haemophilia A. Dr Ri Liesner from Great Ormond Street Hospital for Children, London, UK, presented for the first time the final data on the immunogenicity, efficacy and safety of Nuwiq® (simoctocog alfa) in PUPs followed for up to 100 exposure days.
Dr Liesner, the Principal Investigator of the study, commented that “the NuProtect study has made a valuable contribution to our understanding of factors affecting inhibitor development in haemophilia A and should help to inform treatment decisions in PUPs”.
When treating PUPs with severe haemophilia A, the development of neutralising antibodies against FVIII is the most significant concern. In 105 patients in the NuProtect study, the cumulative incidence of high-titre inhibitors was 17.6% and the cumulative incidence of all inhibitors was 27.9%. Dr Liesner discussed these results in the context of data from the SIPPET study, in which the cumulative incidence of high-titre inhibitors was 28.4% in patients treated with recombinant FVIII products derived from hamster cell lines, and 18.6% in those treated with plasma-derived FVIII products.
Interestingly, an analysis of the underlying F8 gene mutation demonstrated that no patients with non-null mutations developed inhibitors to Nuwiq® in the NuProtect study.
“The data suggest that Nuwiq® has the potential for low immunogenicity in PUPs, with a profile more similar to plasma-derived FVIII products than to recombinant FVIII products derived from hamster cells”, commented Dr Liesner. “This relatively large clinical data set provides support for the use of Nuwiq® in PUPs”.
The data also demonstrated the efficacy and safety profiles of Nuwiq® for the prevention and treatment of bleeding in PUPs. Efficacy in the prevention of spontaneous bleeding events was rated as “excellent” in all 50 patients receiving continuous prophylaxis with Nuwiq®. On-demand treatment was rated as “excellent” or “good” for 93% of bleeding episodes, and efficacy as surgical prophylaxis was rated as “excellent” or “good” for 95% of 19 rated surgeries. Nuwiq® was well-tolerated, with only one possibly treatment-related serious adverse event (a mild rash requiring hospitalisation) reported by investigators, which resolved and treatment was continued.
Dr Larisa Belyanskaya, Head of IBU Haematology at Octapharma, said that “we are proud to be able to communicate such exciting data for Nuwiq® in this challenging patient group. This landmark study adds to the broad clinical experience with Nuwiq® and represents an important step in demonstrating the value of Nuwiq® for patients with haemophilia A at all clinical stages, from the very first treatment to long-term protection”.
Olaf Walter, Board Member at Octapharma, added that “We believe that the immunogenicity profile of Nuwiq® reflects its human cell line origin, and more broadly the foundations of Octapharma’s approach to using human proteins to improve the lives of patients. These data demonstrate the value of our strategy and show Octapharma’s ongoing commitment to the bleeding disorders community”.
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 Nuwiq®
Nuwiq® (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® 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. Treatment of previously untreated patients (PUPs) with Nuwiq® was assessed in the NuProtect study. In an interim analysis of 66 patients, the cumulative incidence of high-titre inhibitors was 12.8%, and the cumulative incidence of all inhibitors was 20.8%2. Nuwiq® is available in 250 IU, 500 IU, 1000 IU, 2000 IU, 2500 IU, 3000 IU and 4000 IU presentations3. Nuwiq® is approved in 59 countries for use in the treatment and prophylaxis of bleeding in patients with haemophilia A (congenital FVIII deficiency) across all age groups3.
About the NuProtect study
The NuProtect study (NCT01712438) was a prospective, multicentre, multinational, open-label, non-controlled phase III study to assess the immunogenicity, efficacy and safety of Nuwiq® in patients with severe haemophilia A and without any previous exposure to FVIII concentrates or blood products containing FVIII. Patients were treated with Nuwiq® for 100 exposure days or up to 5 years. The study was conducted at 38 sites in 17 countries. A total of 110 patients were enrolled. Of these, 108 patients were treated with Nuwiq®.
About Octapharma
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 2018, the Group achieved €1.8 billion in revenue, an operating income of €346 million and invested €240 million into R&D and in capital expenditures in order to ensure future prosperity. Octapharma employs 8,314 people worldwide to support the treatment of patients in 115 countries with products across three therapeutic areas:
Haematology (coagulation disorders)
Immunotherapy (immune disorders)
Critical care (bleeding management and functional volume replacement)
Octapharma has seven R&D sites and six state-of-the-art manufacturing facilities in Austria, France, Germany, Mexico and Sweden.
1. Lissitchkov T et al. Ther Adv Hematol 2019; doi: 10.1177/2040620719858471.
2. Liesner R et al. Haemophilia 2018; 24:211-20.
3. Nuwiq® Summary of Product Characteristics.
View source version on businesswire.com: https://www.businesswire.com/news/home/20191210005421/en/
Contacts
Octapharma AG:
Octapharma AG International Business Unit - Haematology
Olaf Walter
Olaf.Walter@octapharma.com
Larisa Belyanskaya
Larisa.Belyanskaya@octapharma.com
Tel: +41 55 4512121
Ivana Spotakova
Communications Manager
ivana.spotakova@octapharma.com
Tel.: +41793474607
Permalink : https://www.aetoswire.com/news/final-results-from-the-nuprotect-study-demonstrating-efficacy-of-nuwiqreg-simoctocog-alfa-in-previously-untreated-patients-pups-presented-at-ash-2019/en