MIL-OSI United Kingdom: Efanesoctocog alfa approved to prevent and treat bleeding in children and adults with severe or moderate haemophilia A

The Medicines and Healthcare products Regulatory Agency (MHRA) has today, 14 February 2025, approved efanesoctocog alfa (brand name Altuvoct) to be used to treat and prevent bleeding in patients aged 2 years and above with severe or moderate haemophilia A.

Efanesoctocog alfa, the active substance, is a replacement factor VIII protein. This protein is naturally found in the body and is necessary for the blood to form clots and stop bleeding.

People with severe haemophilia A have undetectable factor VIII and, if untreated, may experience up to about 40 episodes of bleed per year.

This medicine is administered as an intravenously (into a vein).

Julian Beach, MHRA Interim Executive Director of Healthcare Quality and Access, said:

“Patient safety is our top priority, which is why I am pleased to confirm approval of efanesoctocog alfa to treat and prevent bleeding in patients 2 years and above with severe or moderate haemophilia A.

“We’re assured that the appropriate regulatory standards of safety, quality and efficacy for the approval of this new formulation have been met.

“As with all products, we will keep its safety under close review.”

In a study with 159 patients aged 12 and above with severe haemophilia A, weekly injections of Altuvoct as prophylaxis led to 65% patients reporting zero overall episodes of bleed over the course of the year-long study; the remaining 35% had much reduced episodes of bleed. Altuvoct was also used to treat individual bleeds.

In a study involving 74 children under 12 years of age with severe haemophilia A, treatment with efanesoctocog alfa yielded similar results to those in older patients.

Efanesoctocog alfa was therefore considered effective for the prophylaxis and treatment of severe haemophilia A in children aged 2yrs and above.

The company extended the indication to those with moderate haemophilia A by means of a modelling exercise.

Like all medicines, this medicine can cause side effects, although not everybody gets them. Some of the potential side effects include headaches and arthralgia (joint pain).

For the full list of all side effects reported with this medicine, see Section 4 of the PIL or the SmPC available on the MHRA website.

Anyone who suspects they are having a side effect from this medicine are encouraged to talk to their doctor, pharmacist or nurse and report it directly to the MHRA Yellow Card scheme, either through the website (https://yellowcard.mhra.gov.uk/) or by searching the Google Play or Apple App stores for MHRA Yellow Card.   

 ENDS  

Notes to editors   

• The new marketing authorisation was granted on 14 February 2025 to Swedish Orphan Biovitrum AB

• This product was submitted and approved via an international recognition  procedure. 

• More information can be found in the Summary of Product Characteristics and Patient Information leaflets which will be published on the MHRA Products website within 7 days of approval. 

• The Medicines and Healthcare products Regulatory Agency (MHRA) is responsible for regulating all medicines and medical devices in the UK by ensuring they work and are acceptably safe.  All our work is underpinned by robust and fact-based judgements to ensure that the benefits justify any risks. 

• The MHRA is an executive agency of the Department of Health and Social Care. 

For media enquiries, please contact the newscentre@mhra.gov.uk, or call on 020 3080 7651