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Novo Nordisk Files Long-acting Factor IX For Haemophilia B In US
Novo Nordisk has filed its long-acting factor IX, nonacog beta pegol in the US for haemophilia B.
Nonacog beta pegol is a glycopegylated recombinant factor IX with a longer half-life, developed for patients with haemophilia B. Novo said that the drug's half-life is five times longer than standard factor IX products, meaning fewer injections are needed.
Although Novo has long had a presence in haemophilia, it faces competition in both haemophilia A and B. In haemophilia B, Biogen and Swedish Orphan Biovitrum have Alprolix (coagulation factor IX (recombinant)) approved in the EU and the US.
The filing of nonacog beta pegol is based on the results from the PARADIGM clinical trial programme which involved 115 patients with severe or moderately severe haemophilia B. Nonacog beta pegol was found to be efficacious in routine prophylaxis, treatment of bleeding episodes and surgery for adults, adolescents and children.
Nonacog beta pegol appeared to be well-tolerated and no safety concerns were identified.
In phase 3 trials, once-weekly administration of 40 IU/kg nonacog beta pegol maintained factor IX activity levels above 15%, reduced the median annualised bleeding rate to 1.0 and showed a potential to prevent bleeds in target joints. These patients reported a significant improvement in quality of life during the trial.
Novo Nordisk has also filed the treatment in the EU.
Mads Krogsgaard Thomsen, executive vice president and chief science officer at Novo Nordisk, said: "With its high factor activity level, less frequent dosing and reduced bleeding rate, nonacog beta pegol has the potential to improve the quality of life for both patients and their families."
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India's First Gene Therapy Offers New Hope For Haemophilia Patients
BHUBANESWAR: In a significant medical breakthrough, India's first human gene therapy with a lentiviral vector for severe haemophilia-A has been found to be successful with zero bleeding rate in patients. Haemophilia is a serious bleeding disorder caused by blood deficiency of clotting factors (factor VIII and factor IX proteins). It can lead to spontaneous bleeding episodes, joint damage, and a reduced quality of life for patients and their families.
The new gene therapy has shown promising results during the first phase trial on five patients, aged between 22 and 41. The annualised bleeding rate has been observed to be zero in all five patients over a cumulative follow-up of 81 months. Supported by the Department of Biotechnology, this first-in-human gene therapy for haemophilia-A patients, has been developed by Centre for Stem Cell Research (CSCR) at CMC, Vellore.
Results of the study conducted on the patients have been published in the recent edition of New England Journal of Medicine. Dr Alok Srivastava, lead author of the study and head of haematology research unit at St John's Research Institute, Bengaluru, said excellent outcomes were observed in median factor-VIII levels, which were assessed during the treatment, and follow-up for a period of around two years.
"No spontaneous bleeding events occurred in any of the patients during or after the gene therapy. Before the therapy, all patients had reported an annualised bleeding rate of at least 20 events. The results of this clinical study reveal a new opportunity for gene therapy that can be offered to all haemophilia-A patients, possibly at an early age," Dr Srivastava said. Haemophilia is a rare disorder and India has the world's second largest burden with a case load of 1.36 lakh.
Hemophilia-A is the most common form and accounts for about 80-85% of cases. The availability of factor VIII replacement therapy, the cornerstone of hemophilia treatment, is limited and expensive. Dr Rabindra Kumar Jena, bone marrow transplant physician at SCB Medical College and Hospital, Cuttack, said, haemophilia is a bleeding disorder that slows the blood clotting process.
Haemophilia-A is the most common type of the condition affecting one in every 5,000 births, he said. Physicians believe that the new gene therapy developed by CMC Vellore would be a game changer in haematology treatment across the globe. What's gene therapy Unlike conventional treatments that require regular administration of clotting factor concentrates, gene therapy offers a one-time or infrequent intervention. This therapy can enable the body to produce factor VIII autonomously, potentially providing a lifelong solution.
As part of the therapy, stem cells are extracted from the blood of the patient, and after certain gene corrections, the cells are transplanted back with a lentiviral vector. The therapy provided the body with the ability to produce factor VIII, ensuring that there is no need for repeated infusions. "The therapy is a new innovation and proved very effective during the first phase trial. It can drastically reduce the frequency and severity of spontaneous bleeding episodes, by restoring clotting factor VIII levels.
This would improve patients' overall quality of life. The therapy, being developed indigenously, could be more affordable and accessible," said Dr Jena. With successful gene therapy, patients may no longer need to rely on clotting factor concentrates or plasma products, which are often scarce or expensive. "It could also pave the way for more advanced genetic treatments for other rare diseases," Dr Rajeeb Swain, senior scientist at Institute of Life Sciences (ILS), Bhubaneswar, said.
DBT & CSCR Executes India's First Human Gene Therapy For Hemophilia A Patients
Read by: 100 Industry Professionals
New Delhi: Offering hope to around India's 1,36,000 hemophilia A affected patients, the Department of Biotechnology and the Centre for Stem Cell research (CSCR), CMC, Vellore, have successfully executed a first in-Human Gene Therapy with lentiviral vector for Severe Hemophilia A.In the groundbreaking study the researchers observed an annualised zero bleeding rate in all the five subjects enrolled with the production of Factor VIII for a prolonged period of time thus obviating the need for repeated infusions, DBT stated.
"The volunteers were followed up for 6 months after undergoing therapy. Factor VIII activity was observed to correlate with vector copy number in the peripheral blood. The annualised bleeding rate was observed to be zero for all five participants over a cumulative follow-up of 81 months", it added.
The department also informed that, scientists from CSCR undertook development of an alternate system for restoring Factor VIII expression, using a lentiviral vector in place of AAV vector and based on promising preclinical data a first-in-human clinical trial was conducted to evaluate the safety and feasibility of this gene therapy technology for severe hemophilia A involving five participants who received autologous haematopoietic stem cells (HSCs) transduced with lentiviral vector expressing Factor-VIII gene.Haemophilia is a serious bleeding disorder caused by the deficiency of clotting factors, (i.E., Factor VIII and Factor IX proteins), leads to spontaneous bleeding episodes, and a reduced quality of life for patients and their families. Severe Hemophilia A is managed with repeated factor VIII replacement or hemostatic products that stop or prevent bleeding. Prohibitive cost, venous access especially in children, patient acceptance etc are some of the bottlenecks with respect to standard of care Hemophilia therapy.
Gene therapy using viral vectors which introduces a normal copy of the gene has emerged as a paradigm-changing therapeutic option for Hemophilia. In the case of haemophilia, gene therapy provides the body with the ability to produce Factor VIII, so that the need for repeated infusions can be obviated.Subscribe to our newsletter to get latest insights & analysis.
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