An European regulatory panel has recommended approval for GlaxoSmithKline’s Strimvelis, a gene therapy for the treatment of Bubble Boy disease. It would be the first children’s gene therapy approved in Europe if it receives final marketing clearance from the European Commission.
Bubble Boy disease is formally called severe combined immunodeficiency, and Strimvelis is meant to treat one form of the extremely rare ailment in patients who can’t land a suitable bone marrow donor. The condition stems from faulty genes which are inherited from both parents and inhibits the proper production of white blood cells. Left untreated, children with Bubble Boy disease usually die within one year.
Gene therapies tend to be as process-intensive as they are pricey. For instance, Strimvelis works by extracting bone marrow cells from patients, repairing them by adding the missing enzyme, and then reinserting them. The EU panel recommendation was clearly influenced by Strimvelis’ stellar clinical trial results—100% of patients treated in a clinical trial survived the condition after an average follow-up time of seven years.
It’s a method that Glaxo hopes to replicate down the line.
“Going forward, we hope to apply this gene therapy platform technology across other diseases, enabling many more patients to benefit from this innovative treatment approach,” said Patrick Vallance, president of R&D at Glaxo, in a statement.
There is just one other gene therapy with conditional approval in Europe: uniQure’s Glybera, which costs more than $1 million per treatment.
Over on this side of the pond, Spark Therapeutics (ONCE) is closest to securing the first-ever FDA approval for a gene therapy. It had a $161 million IPO at the beginning of 2015.