Patients affected by ADA-SCID have compromised immune systems that leave them unprotected from infection-producing bacteria, viruses, and fungi.
Undiagnosed babies with ADA-SCID usually die before they reach age two due to infections.
SCID newborn screening in most states has allowed detection of ADA-SCID in newborns and has led to early initiation of ADA enzyme therapy and improved outcomes.
Ada-SCID affects both boys and girls and is more difficult to treat than X-SCID.
A bone marrow transplant from a good donor can cure ada-SCID 90pc of the time.
So far, more than 40 ADA-SCID
patients have been treated with autologous ex-vivo lentiviral gene therapy at GOSH and UCLA.
ADA-SCID affects an estimated 15 children per year in Europe and following todays approval, patients with the condition who are referred for treatment will be able to receive the gene therapy at Ospedale San Raffaele in Milan.
Martin Andrews, Head of the Rare Disease Unit, GSK said: Todays approval is the result of many years work with our collaborators in Milan and is the next step towards bringing life-changing treatment to patients with ADA-SCID and their families.
To date, over 40 ADA-SCID
patients have been treated in clinical trials with autologous ex-vivo lentiviral gene therapy at UCLA, Los Angeles and at the Great Ormond Street Hospital (GOSH) in London, UK.
The gene therapy for the treatment of ADA-SCID
was originally developed by Ospedale San Raffaele (OSR) and Fondazione Telethon (Telethon), through their joint San Raffaele Telethon Institute for Gene Therapy (SR-TIGET) and was taken forward by GSK through a strategic collaboration formed in 2010 between GSK, OSR and Telethon.
is a very rare and life-threatening disease that affects approximately 350 children worldwide.
is a very rare and life-threatening disease caused by the alteration of a single gene and affects approximately 350 children worldwide.