The Danish multinational Novo Nordisk has presented the results of its phase 3 study of a new haemophilia treatment at the International Society of Thrombosis and Haemostasis Annual Congress in London. The Pharma Times reports that the data showed very promising results, with a significant reduction in spontaneous and traumatic bleeds in patients.
People with haemophilia have a reduced ability to form blood clots, and therefore are more vulnerable to dangerous levels of blood loss. It’s a rare genetic condition, and there are two different types: haemophilia A and haemophilia B, which refers to the clotting factor, but the essential clinical features are the same.
The majority of patients are male, because it is linked to the X chromosome. In mild cases, bleeding follows the normal triggers such as injury, tooth extraction, and surgery. In moderate cases, the bleeding may also be triggered with slight injuries, and sometimes spontaneously.
However, in severe cases, spontaneous bleeding into joints and muscles can occur, which can cause painful swelling, and may eventually lead to arthritis developing. Patients with severe haemophilia require careful clinical management. This is usually in the form of frequent injections of Prophylaxis to provide the missing clotting factor.
The new treatment may be suitable for patients who do not respond well to current treatments, or who wish to avoid intravenous injections. It can be administered in an under-the-skin method to treat both A and B type haemophilia.
Dr Victor Jiménez-Yuste, explorer7 lead investigator, said: “One of the most critical complications in the treatment of haemophilia is the development of inhibitors, as they render standard replacement therapy ineffective and severely limit treatment options for haemophilia B.”
He added: “Based on the results of the explorer7 study, there is a potential for concizumab to become a new treatment option for people living with haemophilia A or B with inhibitors.”
Martin Lange, executive vice president and head of development at Novo Nordisk, said:
“The treatment of haemophilia is complex and no one treatment fits all.”
He added: “Concizumab offers the potential for everyday protection for people living with haemophilia and provides an important potential addition to our haemophilia offering, especially in the haemophilia B with inhibitor population who currently have limited treatment options.”
There is also a new gene therapy called Roctavian which has been recommended for a licence by the European Medicines Agency, Heamaphilia.org reports. It is suitable for treating patients with Haemophilia type A. However, there are no reports that the treatment is being reviewed by the UK regulatory body, NICE.
Gene therapy is still in the early stages, and it is not yet known if it will work safely and effectively on a long-term basis. The treatments have not yet been tested on children, and some adult patients have shown serious side effects, including liver toxicity. Some patients also proved to be unresponsive to the gene therapies, or only had a limited response.
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