Is $3.5 Million a Fair Price for a Lifesaving Gene Therapy?

Gene therapies have the power to cure serious, even fatal, diseases. Yet what captures public attention is often not the transformative effects but the enormous price tags. 

At $3.5 million, Hemgenix, the new gene therapy for hemophilia B, has recently been named the most expensive drug on the planet, unseating another gene therapy, Skysona.

Hemgenix is record-setting, but hardly an anomaly. Skysona, a treatment for a rare neurological disorder, launched at $3 million in September 2022. Zynteglo, a gene therapy for a genetic blood disorder, debuted just one month earlier at $2.8 million. In 2019, Zolgensma was priced at $2.1 million as a treatment for spinal muscular atrophy, a fatal genetic disease affecting infants and young children. Several other treatments land in the hundreds of thousands. 

Yet the remarkable results lead some to call gene therapy a relative bargain. These drugs have the potential — in some cases, the proven ability — to cure illness with a single dose. This liberates patients from the physical, emotional, and financial burden of living with a serious disease, often one requiring highly expensive treatments. 

That's because gene therapy does not treat symptoms. It targets the cause, the genetic defect behind a disease, swapping out faulty code or even inserting a gene that's missing. Sometimes, this happens in a petri dish, and the healthy cells are transferred to the patient. Other times a vector, usually a virus, delivers the genetic material to the patient's cells.

Source: MEDspace