A technology that could offer people with haemophilia B an implant providing long term Factor IX synthesis has been awarded Advanced Therapy Medicinal Product (ATMP) status by the European Medicines Agency’s Committee for Advanced Therapies.
ATMPs are medicines for human use that are based on genes, tissues or cells that ‘offer ground breaking new opportunities for the treatment of disease and injury’. Designated products are eligible for a streamlined regulatory process and additional support from the EMA.
The award has been made to SIG-003, an ‘immune-privileged living therapeutic implant’ developed by Sigilon Therapeutics. The product uses an implant made from bio-inert Afibromer technology to house cells that have been genetically modified to synthesise Factor IX. The implant protects the cells from attack by the patient’s immune system. Press reports suggest an implant may be effective for up to one year.