Despite being one of the most expensive drugs in the world, GlaxoSmithKline's gene therapy Strimvelis won the backing of the often stingy U.K. cost watchdogs to treat a rare disease called ADA-SCID, or "bubble boy" syndrome. The drug, which costs €594,000 (about $700,000), is a one-time treatment that the overseers said is worth the cost.
Strimvelis comes with a "cost that provides value for money in the context of a highly specialised service," NICE reviewers wrote in the draft guidance (PDF). They said it's an "important development" in treatment for the ultrarare disease.
ADA-SCID is a serious immune condition that can kill children before school age if untreated. Patients with the condition are susceptible to recurrent infections and other severe symptoms. For treatment with Strimvelis, teams collect patients' bone marrow cells, then modify and reinject them. It's a one-time procedure that experts believe offers lifelong benefits, according to NICE documents.
NICE reviewers recommended the therapy for patients when no suitable stem cell donor is available. The gene therapy has benefits beyond standard cost-effectiveness measures, according to NICE.
"Because Strimvelis reconstitutes the immune system, it could enable children with the condition to be educated at school and for carers of people with the condition to return to work," the document stated.
According NICE, GSK assumed one person per year would get treatment, but that could vary year to year. On Monday, a GSK spokesperson said the company is "really pleased to see the draft guidance and hope this decision will become final guidance in the next few weeks."
"Strimvelis is the first corrective gene therapy for children to be approved anywhere in the world, and this draft NICE guidance will be encouraging news for those families in the UK who have a child with ADA-SCID and no suitable matched stem cell donor," he added.
But the decision comes even as GSK reconsiders its future in rare diseases. Back in July, the company disclosed that it's exploring a sale for its rare disease portfolio, which includes Strimvelis and two other programs in development.
A spokesperson said at the time that while GSK has “been heavily invested in this area, we believe there is someone else who can best ensure the commercial availability of these medicines for patients.” GSK isn't leaving "cell and gene therapy R&D, but will focus on oncology and more common indications," she added. The company said its immediate focus is "meeting our obligations to patients."
When GSK announced that decision, just two patients had received treatment with Strimvelis, and another two were slated for treatment. Despite their ability to improve patients' lives, such rare disease drugs don't always perform well commercially, and reimbursement can be tough to secure.
The first gene therapy in Europe, uniQure's Glybera, didn't meet sales requirements to justify expenses, that drugmaker previously said as it announced it would not seek to renew a marketing authorization.
Across the pond, the cell and gene therapy market is now set for a test in the U.S. as two CAR-T medications from Novartis and Gilead have won approval, carrying $475,000 and $373,000 price tags before discounts, respectively. Novartis introduced a money-back guarantee for certain patients who receive its drug, Kymriah.