European regulators approve GSK's Strivelis therapy for children with combined immunodeficiency
01 June 2016
European regulators have given the nod to UK drug firm GlaxoSmithKline (GSK) to produce the world's first gene therapy treatment for children.
GSK received the approval by the EC to provide the treatment to children with a rare immune disorder - which could be fatal for affected children.
The company would produce Strimvelis, the first stem cell gene therapy to treat patients with severe combined immunodeficiency caused by adenosine deaminase deficiency (ADA-SCID).
The treatment uses stem cells extracted from the child's own bone marrow, which are then tweaked with DNA for a working copy of the enzyme introduced.
These stem cells are then reintroduced to the patient, whose body would then be able to produce their own working copies of the immune enzyme.
Trial data had shown a 100 per cent survival rate for children receiving Strimvelis up to three years after the treatment.
The condition results from the inability of the body to produce a key enzyme in maintaining the immune system, leading to the build-up of toxins.
Consequently, children affected by the disease are unable to fight off everyday infections.
Around 15 children in Europe are affected by ADA-SCID each year.
A gene therapy treatment has been approved for production by a major pharmaceutical company for the first time, in what scientists hope will open up a long-promised field of medicine.
GlaxoSmithKline will be allowed to sell a genetic therapy to treat an extremely rare immune disorder in children. The treatment works by taking cells from the child's own body, replacing a defective gene and then reimplanting them.
In a press release GlaxoSmithKline said, ADA-SCID affects an estimated 15 children per year in Europe and following today's 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: ''Today's 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. This is the start of a new chapter in the treatment of rare genetic diseases and we hope that this therapeutic approach could also be used to help patients with other rare diseases in the future.''