Going into a two-day FDA advisory committee meeting this week, bluebird bio faced uncertainty over the viability of its gene therapy prospects. But the drugmaker has impressed an FDA panel and come away with a pair of expert endorsements for its lead drugs.
In a Friday afternoon vote, the FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee voted 13 to 0 that the benefits of bluebird’s betibeglogene autotemcel (beti-cel) outweigh its risks. The drug is under review at the FDA to treat adolescent and pediatric patients with the rare blood disorder beta thalassemia who require regular red blood cell transfusions.
After the vote, panel member Jeannette Lee, Ph.D., said the efficacy data for the medicine look “outstanding.” She said it could be “life-changing” for patients to be able to achieve independence from blood transfusions after receiving the drug.
The drug is set for an Aug. 19 decision deadline at the FDA. The agency doesn’t always follow its advisory panel votes, but it typically does.
The Friday vote followed another favorable outcome on Thursday for bluebird's elivaldogene autotemcel, also known as eli-cel. That drug is under FDA review in cerebral adrenoleukodystrophy and also scored unanimous backing from the panel.
Heading into the meeting, FDA reviewers seemed positive on beti-cel but flagged safety concerns for eli-cel. During Thursday’s meeting, the panelists said they believe the drug is an appropriate treatment option for patients despite the potential risks.
Bluebird is no doubt celebrating the outcome of the meeting. Over the last couple of years, the company has faced commercial setbacks in Europe and regulatory and clinical delays in the U.S., but it could now be nearing a launch in the country.
Beti-cel won approval in Europe in 2019 under the brand name Zynteglo, where it was priced at almost 1.58 million euros (near $1.8 million) based on the average 2019 exchange rate. However, the company pull the drug off the market after it couldn’t convince payers there of the costly therapy's value. In April, the company cut 30% of its staff.
In the U.S., drug cost watchdogs at the Institute for Clinical and Economic Review have endorsed the drug at a potential one-time price of $2.1 million.
Aside from beti-cel and eli-cel, bluebird is also advancing lovotibeglogene autotemcel (lovo-cel) in sickle cell disease. The company is targeting roughly 1,500 patients with beti-cel but could ultimately reach 20,000 with lovo-cel if that drug scores FDA backing. Bluebird plans to submit lovo-cel to the FDA early next year.