FDA approves most expensive drug in history

John Hugh DeMastri, DCNF

The U.S. Food and Drug Administration (FDA) on Wednesday approved a gene therapy for a rare blood disease which is set to reach the market at a record $2.8 million for a single dose, according to a press release by the therapy’s creator, Bluebird Bio.

Beta-thalassemia is an inherited blood disorder that causes a patient’s blood to fail to circulate oxygen through the body, according to the FDA press release concerning the approval. Bluebird’s new therapy, Zynteglo, infuses patients with cells that have a working copy of the gene responsible for the disorder, allowing the patient to produce blood that functions properly, according to a Bluebird press release.

“[Zynteglo] has the potential to untether from the health care system patients who, on average, currently require transfusions every two to five weeks for life and may lose decades of life relative to the general population,” Bluebird Chief Communications Officer Sarah Alspach told the Daily Caller News Foundation. “We took this profound impact into consideration when pricing the therapy.”

Patients with beta-thalassemia typically require regular blood transfusions, which can lead to heart and liver problems when administered repeatedly, according to the FDA. Zynteglo is administered as a single dose therapy, and of 41 patients in clinical trials, 89% were able to forego transfusions, the FDA reported.

Lifetime care for beta-thalassemia can cost up to $6.4 million in the U.S., and patients spend 23 times the national average per year on medicine, Bluebird argued in a press release.

Until Wednesday, the most expensive drug approved by the FDA was fellow gene therapy Zolgensma, which treats a rare childhood muscle disease, at $2.1 million for a one-time dosage, GoodRx reported. Two children recently died following treatments of Zolgensma due to liver failure, the first fatalities associated with the drug, according to Genetic Engineering & Biotechnology News.

Zynteglo is approved for use in both children and adults who are reliant on blood transfusions, according to the Bluebird press release.

“We are ushering in a new era in which gene therapy is no longer a tool reserved only for severe childhood diseases but now has the potential to transform existing treatment paradigms for chronic diseases that carry a lifelong burden of care,” Alspach told the DCNF.

Bluebird has had mixed success in Europe, where Zynteglo was rejected at a price point of $1.8 million by regulators responsible for price negotiations, Forbes reported in May. Negotiators for the British National Health Service said that the benefits did not justify the price, but the U.S. Institute for Clinical and Economic Review, an independent pricing watchdog, said that Zynteglo would be cost effective at $2.1 million, assuming a five year payment period and robust follow-up care, Forbes reported.

The FDA referred the DCNF to Wednesday’s press release.

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