NICE recommends Altuvoct as NHS treatment option for hemophilia A
Decision will make therapy available through UK's public healthcare system
The U.K.’s National Institute for Health and Care Excellence (NICE) has recommended Altuvoct (efanesoctocog alfa) be available through the National Health Service (NHS), England’s public healthcare system, as a treatment option to manage and prevent bleeds in people with severe hemophilia A, ages 2 and older.
This decision came shortly after the treatment’s approval last month by the country’s Medicines and Healthcare products Regulatory Agency. The therapy already was approved in the European Union under the same brand name. It’s also been approved in the U.S., where it’s marketed as Altuviiio.
“[This] decision … shows how, by collaborating with system partners we can quickly recommend treatments that deliver tangible benefits for patients and the NHS alongside value-for-money for taxpayers,” Helen Knight, director of medicines evaluation at NICE, said in an institute press release.
According to NICE, Altuvoct, with its weekly dosing regimen, offers patients an alternative to other available therapies that are given several times per week.
“Current factor VIII replacements can be difficult to manage due to the need for frequent dosing to prevent potentially life-threatening and debilitating bleeding episodes. … [Altuvoct] only has to be taken once a week,” Knight said. “Combined with its effective bleeding control, it has the potential to have a significant positive impact for some people with severe [hemophilia] A.”
Altuvoct to be available through NHS as on-demand and preventive treatment
James Palmer, NHS England’s medical director for specialized services, called the decision “an important step forward in treatment for patients,” saying it “ensures they continue to have access to the latest advances in care on the NHS to help prevent bleeding episodes.”
Hemophilia A is caused by mutations in the F8 gene that lead to a lack or dysfunction of a protein called factor VIII, or FVIII, which is crucial in the blood clotting process. This typically results in episodes of excessive bleeding and easy bruising that may occur spontaneously or as a result of injury or trauma.
Standard treatment generally involves replacement therapies, which supply a version of the missing or dysfunctional FVIII to patients to treat or prevent bleeds.
Altuvoct is a new type of hemophilia A replacement therapy that contains a lab-made version of FVIII fused to a fragment of von Willebrand factor — another clotting protein that helps stabilize and prevent FVIII degradation. In addition, the therapy has two other modifications that help it stay active in the body for longer periods of time.
Together, these features enable Altuvoct to have a more sustained presence in the bloodstream, and therefore be administered less frequently when used as a prophylactic treatment, or to prevent bleeds.
The therapy, when used as a prophylaxis, is recommended to be given once weekly at a dose of 50 international units per kilogram of body weight, via an intravenous, or into-the-vein, injection. It also may be used as an on-demand therapy for active bleeds, including those occurring during surgery.
It’s fantastic news that this time-saving therapy will now be available to help improve patients’ lives, reinforcing the NHS’ commitment to providing innovative treatments at value for the taxpayer.
The therapy’s approval in the U.K. was based on data from the Phase 3 XTEND-1 trial (NCT04161495), which enrolled 159 people with severe hemophilia A. Two U.K. sites were among the study’s 47 locations.
The results showed that 65% of patients who received weekly injections of Altuvoct had no bleeding episodes over the course of one year of treatment. Altuvoct also eased pain and effectively treated bleeding episodes when used on demand.
Similar results were seen in the Phase 3 XTEND-Kids trial (NCT04759131), which also included the two U.K. study sites. That trial enrolled children younger than 12 with severe hemophilia A.
“This condition can have really debilitating and painful impacts and it’s fantastic news that this time-saving therapy will now be available to help improve patients’ lives, reinforcing the NHS’ commitment to providing innovative treatments at value for the taxpayer,” Palmer said.
About the Author
