Oncolytics Biotech Inc T.ONC

bluebird's gene therapy drug most expensive in the world

[Noteable]

The company is charging $3 million per treatment with Skysona, higher than Zynteglo’s $2.8 million, making it the priciest therapy in the world.

Skysona is bluebird’s second gene therapy after Zynteglo in the U.S. Previously, the biotech had to pull both products in Europe and folded commercial activities in the region after failing to reach reimbursement agreements with local governments.

The FDA handed out the accelerated approval based on data from two small clinical trials. The drug was found to be better than natural history data at preventing the emergence of functional disabilities or death after two years.

Right before granting the approval, the FDA also lifted a clinical hold that it previously issued in August 2021. The hold was in place after a patient who got Skysona developed myelodysplastic syndrome (MDS), a type of blood cancer. Altogether, three patients in Skysona’s clinical program showed MDS, which is believed to be caused by the Lenti-D viral vector that bluebird uses to deliver a functional ABCD1 gene into the human genome.

https://www.fiercepharma.com/pharma/3m-gene-therapy-bluebird-breaks-own-record-fda-approval-skysona