Treatment

EMA’s human medicines committee (CHMP) adopted a positive opinion, recommending the granting of a marketing authorisation for the medicinal product Hympavzi¹, 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.

The applicant for this medicinal product is Pfizer Europe Ma EEIG.

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 eight medicines for approval at its May 2024 meeting.

A recommendation for a conditional marketing authorisation was made for Durveqtix (fidanacogene elaparvovec), 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. See more details in the news announcement in the grid below.

More information here

EMA’s human medicines committee (CHMP) recommended eight medicines for approval at its April 2024 meeting.

The CHMP recommended granting a marketing authorisation for Altuvoct* (efanesoctocog alfa), for the treatment and prophylaxis of bleeding in patients with haemophilia A, a rare inherited bleeding disorder caused by lack of factor VIII.

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:  https://www.ema.europa.eu/en/medicines/human/EPAR/hemgenix#:~:text=information%20on%20Hemgenix-,Overview,blood%20clots%20to%20stop%20bleeding

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: 

https://www.ema.europa.eu/en/documents/overview/roctavian-epar-medicine-overview_en.pdf