Hemgenix® (etranacogene dezaparvovec) has been approved by the Italian Medicines Agency (AIFA). This decision marks a key milestone in access to therapeutic innovation in Italy.
Hemgenix was the first gene therapy to be approved in Europe for the treatment of severe and moderately severe haemophilia B in adult patients without a history of Factor IX inhibitors.
Please find the press release attached, as well as further information summarised below:
- Eligible patients will now have the option to access Hemgenix in Italy, as the AIFA recognised Hemgenix as an innovative therapy.
- Haemophilia B occurs due to a gene mutation which causes coagulation Factor IX (FIX), an important clotting protein, to be deficient or missing. There is no cure for haemophilia and people living with the disease need life-long treatment.
- An estimated 5,900 people live with haemophilia B in Europe. Those affected are particularly vulnerable to bleeds in their joints, muscles, and internal organs, leading to pain, swelling, and joint damage.
- Current treatments for moderate to severe haemophilia B include life-long prophylactic infusions to replace or supplement low levels of FIX, with scheduled infusions carrying a significant burden for patients, carers, and their families.
- Patients in France, Denmark, Austria, Spain, the UK and Germany have already experienced treatment with Hemgenix.
This milestone follows in the footsteps of groundbreaking reimbursement and access agreements reached in Spain, Switzerland, Germany, the UK (including Scotland), Denmark and Austria, paving the way for other European countries and beyond to implement innovative access solutions for gene therapies.
