Expensive gene therapy receives its first patient in a commercial treatment

Known as Strimvelis, the therapy treats a rare inherited immune deficiency by fixing a problem within the patient’s DNA.

Gene therapy has been used extensively in clinical trials but has had a slow start commercially. This is only the second commercial use of gene therapy, the first of which was with a drug called Glybera in 2015.

Gene therapies are relatively complex compared to standard pills, often requiring invasive procedures. The conditions they treat are also exceptionally rare — each year only about fifteen children in Europe and twelve in the United States are affected by ADA-SCID, the disease Strimvelis treats. Due to their complexities, the drugs are also some of the most expensive in the world. Strimvelis is priced at $648,000. Glybera was listed at $1 million. Uniqure, the company behind Glybera, announced last month that it would remove its drug from the market due to lack of demand.

The project lead for Strimvelis, Jonathan Appleby, told Technology Review that the year-long delay between European approval and treatment was due to cross-border reimbursement for the therapy, which is only available in Milan, Italy.

“It’s definitely a bad sign for patients,” said Casey Quinn, a health economist with a focus in European drug pricing. “It remains to be seen whether this represents some kind of watershed, or it will take just as long to go from one [patient] to two?”

Strimvelis treatment is complex, necessitating a “specialized environment,” according to Lucia Monaco, chief scientific officer at Fondazione Telethon, the institution that initially developed the therapy. During the procedure, doctors remove cells from a patient’s bone marrow. The cells are modified externally before being replaced through an infusion into the patient’s veins.

Though commercial use has been slow for gene therapies, many patients continue to undergo treatment through clinical trials. Spark Therapeutics, a United States-based biotech company, is scheduled to request approval for commercial sale of a gene therapy that treats a condition that causes blindness later this year.