UK approves once-weekly treatment for severe Hemophilia A

U.K. – People with severe haemophilia A in the UK now have access to a new, long-acting Factor VIII replacement therapy that requires fewer injections.

Altuvoct (efanesoctocog alfa), developed by Sobi and Sanofi, has been recommended for NHS use by the National Institute for Health and Care Excellence (NICE).

Current Factor VIII replacement therapies require injections two to three times a week to prevent dangerous bleeding episodes.

In contrast, Altuvoct is administered just once a week, making it a more convenient option for patients.

The UK Medicines and Healthcare products Regulatory Agency (MHRA) approved Altuvoct on February 14, following strong clinical trial results.

According to NICE’s final guidance, Altuvoct will be available for people aged two and older as an alternative to existing treatments.

In clinical trials, 65% of patients had zero bleeding episodes over a year with weekly injections, while the remaining 35% had a significant reduction in bleeding events.

Impact on patients and the market

There are about 7,700 people with heemophilia A in England, and around 1,900 of them have severe disease.

For these patients, frequent injections can be challenging and time-consuming. NICE’s Helen Knight highlighted that Altuvoct’s effective bleeding control and reduced dosing frequency could greatly improve patients’ quality of life.

The Heemophilia Society welcomed the approval, expecting the final confirmation in March and NHS availability in July.

The therapy should also become accessible in Wales, Scotland, and Northern Ireland soon after.

Kate Burt, CEO of the Haemophilia Society, emphasized that Altuvoct’s less frequent dosing would allow patients to focus more on their daily lives rather than managing their condition.

Competition and market growth

Altuvoct replaces Eloctate (efmoroctocog alfa), an older haemophilia A therapy from Sobi and Sanofi that required injections every four days or more.

However, Eloctate sales have dropped due to competition from Roche’s Hemlibra (emicizumab), an antibody-based treatment that has a broader approval and generated US $5 billion in sales last year.

Despite this, Altuvoct—sold as Altuviiio in the US—is gaining traction. Sales surged 330% last year to US $712 million, mostly in the US market.

Analysts at Barclays predict that global sales could peak at US $2.4 billion as the therapy expands into Europe and other regions.