Give your opinion on a genetically modified medicine for the treatment of hemophilia B

date: 28/02/2024

The FPS Public Health and the FAMHP invite you to participate in the public consultation on a clinical trial of the genetically modified medicine PF-06838435  for the treatment of hemophilia B. The public consultation runs from 28 February 2024 to 28 March 2024.

For each clinical trial application for the use of a genetically modified organism, the government organises a thirty-day public consultation, in accordance with the Royal Decree of 21 February 2005. As a citizen, you can give your opinion on this clinical trial application with a genetically modified medicine.

Patients with haemophilia B are deficient in the factor IX (FIX) protein that is needed to form a blood clot to stop bleeding. This deficiency is caused by a fault in the gene responsible for the production of a functional protein. Current treatment for these patients involves administering blood coagulation factor IX. 

In this clinical trial, the medicine PF-06838435 (fidanacogene elaparvovec) will be administered once via intravenous infusion to adult male patients with moderately severe to severe haemophilia B. These patients have previously participated in a lead-in study in which they received blood coagulation factor IX replacement therapy according to their usual care.

The medicine PF-06838435 has been developed to deliver a corrected version of the factor IX gene to allow the patients to produce a functional FIX protein. All patients will be followed up for six years after the administration of PF-06838435.

The aim of this trial is to assess the safety and efficacy of the medicine PF-06838435.

The clinical trial will take place at the University Hospital Saint-Luc in Sint-Lambrechts-Woluwe.

How to give your opinion?
The public consultation runs until 28 March 2024 inclusive. You can access the various data in the application file, as well as an online form to send your comments or ask your questions.

General information on GMO - public consultation.

Last updated on 28/02/2024