Efanesoctocog Alfa Approved to Prevent and Treat Bleeding in Children and Adults with Severe or Moderate Haemophilia A
London, UK – February 14, 2025 – The UK Medicines and Healthcare products Regulatory Agency (MHRA) has approved efanesoctocog alfa (Eloctate) for the prevention and treatment of bleeding episodes in children and adults with severe or moderate haemophilia A.
Haemophilia A is a rare genetic bleeding disorder caused by a deficiency or abnormality of the clotting factor VIII (FVIII). People with haemophilia A experience prolonged or spontaneous bleeding episodes that can be life-threatening.
Efanesoctocog alfa is a recombinant porcine FVIII (rpFVIII) product that is produced using a proprietary cell culture technology. It is designed to have a prolonged half-life in the body, which may reduce the frequency of bleeding episodes and the need for frequent infusions.
The approval of efanesoctocog alfa is based on the results of two clinical trials: A-LONG and Kids A-LONG. In the A-LONG trial, which involved 141 adults and adolescents with severe haemophilia A, efanesoctocog alfa was shown to be effective in preventing and treating bleeding episodes. The trial demonstrated a significant reduction in the annualized bleeding rate (ABR) and the number of infusions required per year.
In the Kids A-LONG trial, which involved 55 children with severe haemophilia A, efanesoctocog alfa was also shown to be effective in preventing and treating bleeding episodes. The trial demonstrated a significant reduction in the ABR and the number of infusions required per year.
“The approval of efanesoctocog alfa is a significant advance in the treatment of haemophilia A,” said Professor Ian Peake, Director of the Haemophilia Centre at the Royal Free Hospital in London. “This new treatment has the potential to improve the quality of life for people with haemophilia A by reducing the frequency of bleeding episodes and the need for frequent infusions.”
Efanesoctocog alfa is administered subcutaneously (under the skin) once weekly. The recommended dose is based on the patient’s weight and response to treatment.
The most common side effects of efanesoctocog alfa are injection site reactions, headache, and nausea. Serious side effects are rare.
Efanesoctocog alfa is now available in the UK.
Efanesoctocog alfa approved to prevent and treat bleeding in children and adults with severe or moderate haemophilia A
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