Gene therapy pioneer bluebird bags a 'breakthrough' title for lead program

bluebird's David Davidson

Bluebird bio nabbed the FDA's breakthrough drug title for LentiGlobin BB305 as a new treatment for beta-thalassemia major, putting one of the industry's top experimental gene therapies back squarely in a promising spotlight. The biotech put out the news alongside Roche's announcement that its PD-L1 checkpoint inhibitor MPDL3280A picked up its second breakthrough drug designation, this time for non-small cell lung cancer.

Bluebird's ($BLUE) BTD win helps solidify its rep as one of the leading gene therapy developers in biotech. The biotech believes that the scientific roller coaster ride in gene therapy work is over and that its lead therapy for beta-thalassemia and sickle cell diseases will prove that you can safely insert a corrective gene into patients. And their approach has drawn some positive attention at the agency.

"Our early clinical data investigating the use of LentiGlobin in patients with multiple genotypes of beta-thalassemia major, including beta-0/beta-0, the most severe genotype, are very encouraging, and we remain on track to complete enrollment in the Northstar and HGB-205 studies in 2015," says Dr. David Davidson, chief medical officer of bluebird bio, in a statement. "In light of the breakthrough designation, we look forward to working even more closely with the FDA to expedite the development of LentiGlobin for the treatment of beta-thalassemia major."

Cambridge, MA-based Bluebird has been testing an improved lentiviral vector needed to get a corrective beta-globin gene in place to repair patients' ability to produce hemoglobin and wean them off the blood transfusions needed to stay alive.

Just last December bluebird noted in preliminary results presented at the annual the American Society of Hematology meeting that the first four patients treated with bluebird's therapy remain transfusion-free after at least three months follow up, affirming the treatment's promise as a functional cure for the disease. That update added two more transfusion-free patients to the two bluebird disclosed over the summer, news that sent its shares up more than 50% in June.

Roche, meanwhile, added boasting rights for a second BTD on its PD-L1 drug, which had earlier won a nod for bladder cancer. The pharma giant has been pushing along its checkpoint inhibitor, designed to unleash an immune system attack, behind the lead drugs from Bristol-Myers Squibb and Merck, which are both pushing hard on lung cancer programs of their own. Lung cancer is considered a major market opportunity for the leading players in this field, and Roche has been mixing and matching cancer therapies for an ambitious in-house program.

Over the last two years the FDA has handed out dozens of these breakthrough designations to drug developers, promising to work closely with researchers to help speed trial development and advance cutting-edge therapies to a final marketing decision. Over that time a number of developers have noted that the FDA's new program has helped accelerate their work, shaving time out of the process. And several of these drugs in cancer have gone on to accelerated approval.

- here's the release from bluebird
- here's the release from Roche

(FierceBiotech Senior Editor Damian Garde contributed to this report.)

Special Reports: 2012 Fierce 15 - bluebird bio | The top 10 biotech IPOs of 2013 - bluebird bio

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