Gene Therapy for ‘Bubble-Boy Disease’ Recommended for UK Approval

UK drug regulators granted preliminary approval to GlaxoSmithKline’s gene-therapy treatment Strimvelis for patients with adenosine deaminase deficiency–severe combined immunodeficiency (ADA–SCID), also known as “bubble-boy disease,” when no suitable human leukocyte antigen-matched related stem-cell donor is available.

The approval, if finalized after comments close on the recommendation Nov. 13, would make the treatment eligible for coverage under the National Health Service. The estimated cost of the medication is $700,000 for a single infusion, intended as a lifetime cure. The treatment is available only at a single facility in Italy, and additional costs would be generated by transportation and in-patient stays.

The regulators made their recommendation while noting concerns about the size and certainty of clinical trials.