Treatment

In a report on the third quarter 2025 company’s results and corporate updates, BioMarin announced that it pursues options to divest Roctavian and remove it from their portfolio. BioMarin believes that Roctavian “has an important role to play in the treatment of hemophilia A”, therefore the company is evaluating “out-licensing options” for this gene therapy.

Read more here.

On May 12, 2025, the European Medicines Agency (EMA) published on its website the information about a supply shortage with NovoSeven  (eptacog alfa) in some EU countries.

The supply shortage or limited availability affect some of the Member States where the medicine is marketed, including Belgium, Denmark, France, Germany, Luxembourg and Sweden; in some Member States, English-labelled packs for certain doses will be available until the end of 2025.

Recommendations for patients and carriers:

• Due to manufacturing issues, there are supply shortages or limited availability of NovoSeven in some EU countries.
• If NovoSeven is not available, your doctor may prescribe a different medicine.
• When you receive NovoSeven, it may be accompanied by information in English (on the box and package leaflet). If you need help understanding the information or have any other questions, speak to your doctor or pharmacist.
• For additional information about the supply of NovoSeven, consult your country’s shortage register or contact your national competent authority.

The EHC will keep monitoring closely the situation with availability of NovoSeven in its member countries.

Pfizer will end global development and commercialisation of its haemophilia gene therapy Beqvez.

As stated by several media, including, Reuters, on February 20, 2025, Pfizer said it would stop global development and commercialisation of its haemophilia gene therapy Beqvez.

Pfizer Inc.  announced  that the European Commission (EC) has granted marketing authorisation for HYMPAVZI™ (marstacimab, intended for prophylaxis of bleeding episodes in patients aged 12 years and older, weighing at least 35 kg, who have severe haemophilia A or B.

Hympavzi is indicated for routine prophylaxis of bleeding episodes in patients 12 years of age and older, weighing at least 35 kg, with:

• severe haemophilia A (congenital factor VIII deficiency, FVIII < 1%) without factor VIII inhibitors, or
• severe haemophilia B (congenital factor IX deficiency, FIX < 1%) without factor IX inhibitors.

Read more here 

EMA’s human medicines committee (CHMP) recommended granting a marketing authorisation for Alhemo (concizumab), a medicine for routine prophylaxis of bleeding in patients with haemophilia A or B with inhibitors, two types of a rare inherited bleeding disorder.

The applicant for this medicinal product is Novo Nordisk A/S.

Alhemo is indicated for routine prophylaxis of bleeding in patients with:

• haemophilia A (congenital factor VIII deficiency) with FVIII inhibitors and of 12 years of age or more.
• haemophilia B (congenital factor IX deficiency) with FIX inhibitors and of 12 years of age or more.

Read more here.

Beqvez (previously Durveqtix (fidanacogene elaparvovec)) receives a conditional marketing authorisation valid throughout the EU on 24 July 2024. A new gene therapy treatment for haemophilia B, a rare inherited bleeding disorder. This medicine was supported through EMA’s Priority Medicines (PRIME) scheme, which provides early and enhanced scientific and regulatory support for promising medicines with a potential to address unmet medical needs.

More information here

Altuvoct is a medicine used to prevent and treat bleeding in adults and children with haemophilia A. Haemophilia A is an inherited bleeding disorder caused by a lack of factor VIII, a protein that helps the blood clot.

Haemophilia A is rare, and Altuvoct was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 28 June 2019.

Altuvoct contains the active substance efanesoctocog alfa.

More information here

The EHC herewith communicates the following:

• There has been a small number of complaints for the BAXJECT II and BAXJECT II Hi-Flow reconstitution devices that concern the presence of particulate matter before administration.
• This issue is not related with any medicine produced by Takeda (RECOMBINATE, RIXUBIS, ADVATE, ADYNOVATE and FEIBA) or with the diluent used for the reconstitution of these products, but with the device (BAXJECT II) that is used to mix the medicine and diluent, before administration.
• There hasn’t been any presence of particles in the medicine/diluent reported.
• These devices are co-packaged with the medicine and the diluent.
• No adverse events linked to the particulate matter have been reported.
• Takeda has decided, as a precautionary measure, to voluntarily replace the devices manufactured between October 2021 and January 2022.

Full information can be found here https://www.ehc.eu/baxject-ii-baxject-ii-hi-flow-reconstitution-devices/

The first gene therapy for haemophilia B has been given conditional marketing authorisation in Europe by the European Commission on February 20, 2023. 

Hemgenix is a medicine used to treat adults with severe and moderately severe haemophilia B, an inherited bleeding disorder caused by the lack of factor IX (a protein needed to produce blood clots to stop bleeding). It is used in adults who have not developed inhibitors (proteins made by the body’s natural defenses) against factor IX.

Haemophilia B is rare, and Hemgenix was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 21 March 2018. Further information on the orphan designation can be found here: EU/3/18/1999 

Hemgenix contains the active substance etranacogene dezaparvovec and is a type of advanced therapy medicine called a ‘gene therapy product’. This is a type of medicine that works by delivering genes into the body.

Read more on the EMA website here

Roctavian is a medicine for treating severe haemophilia A, an inherited bleeding disorder caused by the lack of a clotting protein known as factor VIII. It is used in adults who do not have inhibitors (antibodies) against factor VIII and who have no antibodies against adeno-associated virus serotype 5 (AAV5). Roctavian contains the active substance valoctocogene roxaparvovec and is a type of advanced therapy medicine called a ‘gene therapy product’. This is a type of medicine that works by delivering genes into the body.

Read more on the EMA website here.