Soon after Bluebird Bio’s Zynteglo got approval from the FDA, the company has gotten FDA approval for Skysona, a drug for the neurological disorder CALD (cerebral adrenoleukodystrophy), which is quite rare. The charge for the treatment is a record-breaking $3M, which surpasses the nearly month-old record of Zynteglo, which was $2.8M.
The payment to be made will not be based on the outcome, which is the case with Zynteglo therapy. The company claims such a method will be challenging for the patients and hard to implement by the company.
Andrew Obenshain, the CEO of the company claims that the price tag replicates the benefit delivered by the treatment as an immediately needed option to decelerate the development of the disease.
This is the company’s second approved therapy in the U.S after Zynteglo. These therapy treatments are expensive because of their ability to treat deadly diseases with a single dose. Prior to these therapies from Bluebird, the priciest drug in the world was Zolgensma from Novartis, priced at $2.1M.
CALD can cause a decline in neurological abilities and patients may end up losing voluntary movement. Without treatment, nearly 50% of the patients, mostly boys, die within half a decade after symptoms are seen. The drug can be used to treat boys between 4 and 17 years of age with active CALD.
By the company’s estimation, about 1 newborn male in 21,000 is affected by ALD in the U.S., and about 40% of those affected, are expected to be CALD, which roughly translates to nearly 40 cases a year in the country.
The FDA approval was given after results from two small trials were submitted to the authority. To confirm Skysona’s effectiveness, the company will continue monitoring the data from the 67 patients treated using the therapy, for a decade and a half, according to Rich Colvin, Chief Medical Officer of the company.
Prior to the approval, FDA also lifted the clinical hold it hads issued a year ago in August 2021. The hold was due to a patient developing MDS (myelodysplastic syndrome), which is a blood cancer type, after treatment with Skysona. A total of three patients developed MDS. The drug comes with a hematologic malignancy warning on the box.
“We are confident that the risks of Skysona will be weighed carefully with the risks of other treatment approaches and the CALD itself, as families and clinicians make these complex and deeply personal treatment decisions,” Obenshain said.
Commercial availability at certain treatment centers is planned for 2022 end. Only Children’s Hospital of Philadelphia and Boston Children’s Hospital will have the treatment initially.
The two priority review vouchers given to Bluebird after two consecutive approvals are planned to be sold by the company for $100M a piece to keep operations going. Multiple clinical holds and the European commercial setback forced the company to restructure back in April.
Jason Cole, Chief Strategy and Financial Officer says that the company believes it has enough resources to effectively commercialize both the drugs, Skysona and Zynteglo as well as to file the biologics application in the first quarter of the next year, on time.