The new drug approved for the inherited coagulation disorder, offering a simpler dosage and increased quality of life.

Alpha efanesoctocog (also called Altuvoct and performed by the Swedish Orphan Biovitrul) It can be administered as an injection once a week instead of some current treatments that are given several times a week.

Affecting in large part of men, hemophilia A is a rare, inherited and incurable condition, which stops the blood from coagulating correctly. People with hemophilia do not have enough coagulation factor called factor VIII in their blood or do not work properly. This means that they cannot form strong clots and thus bleeding more than usual. Blood can be the result of a lesion and, when the condition is severe, bleeding in the joints and muscles can happen without any injury.

Blood can be prevented by factor VIII injections administered every 2 to 3 days. A non-factor treatment VIII, emicizumab, is also available for people of all ages.

About 7,700 people in England have hemophilia A by approximately 1,900 who have severe hemophilia A.

Helen continues: “Severe hemophilia A is a disease inherited throughout life that can endanger life. May affect a person’s ability to perform normal daily activities due to the risk of suffering a bleeding. Current replacements of factor VIII may be difficult to manage due to the need for a frequent dose to prevent the potential bleeding episodes that can endanger life and debilitating.

“Efanesoctocog alpha should be taken only once a week. Combined with its effective bleeding control, has the potential to have a significant positive impact for some people with severe hemophilia A.

A spokesman for the Department for Health and Social Assistance said: “We know that those living with rare diseases and their families are facing huge daily challenges, and through our action plan for rare diseases, we intend to focus their voices to approach the difficulties they face.

“We have hired ourselves at 3 new actions to improve the coordination of care, access to specialized treatments and to strengthen research on innovative therapies for these rare conditions.

“More broadly, our plan for change will turn NHS by chasing waiting lists and investing into quality units to provide all patients – including those with rare diseases – to receive the care and treatment they deserve.”

Professor James Palmer, NHS England’s medical director for specialized services said: “This is an important step in treatment for patients with severe hemophilia and ensures that they will continue to have access to the last progress in NHS care to help prevent bleeding episodes.

“This condition can have truly weak and painful impacts and it is a fantastic news that this time saving therapy will now be available to help improve patient life, strengthening NHS commitment to provide innovative taxpayer treatments.”