GSK promises ‘bubble boy’ gene therapy will not break the bank as new pricing model revealed

GlaxoSmithKline ($GSK) has only just received a thumbs-up from a European Medicines Agency committee for its groundbreaking new gene therapy treatment–but it’s already thinking about pricing as it gears up for a final European approval in the coming months.
The CHMP–the safety and efficacy arm of the European regulator–recommended approval of GSK’s new drug Strimvelis on Friday to treat the incredibly rare and fatal disorder known colloquially as “bubble boy syndrome,” more than a decade after the EMA first granted the drug an orphan designation.
Strimvelis has been developed in partnership with Italy’s San Raffaele Telethon Institute for Gene Therapy and works as an ex vivo gene therapy for severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID)–aka “bubble boy syndrome,” as children with the disorder must be kept protected from any infections due to their highly fragile immune system.