The European Medicines Agency (EMA) recommended approval for a gene therapy named Strimvelis, which is made by GlaxoSmithKline.
Strimvelis is for treating a rare condition called ADA Severe Combined Immune Deficiency (ADA-SCID), reports Reuters. The disease prevents individuals from forming a certain type of white blood cell, which impacts their immune system.
An estimated 15 children are born with this year condition per year in Europe, and survival rates are low. Patients diagnosed with this disorder usually don’t survive more than two years unless a suitable bone marrow transplant is made.
However, Reuters notes the rarity of this disease has made it difficult to find a matching bone marrow transplant.
The drug operates in two phases, writes FierceBiotech. First, it uses a viral vector to target stem cells extracted from bone marrow and to insert healthy versions of the ADA gene into those cells. Next, the patient’s immune system begins repairing itself after the cells are reintroduced into the body.
Researchers saw exceptionally high survival rates in patients participating in the clinical trial with no adverse events reported.
A final decision from the EU is expected within the next few months.
Filed Under: Drug Discovery