Bluebird to seek
approvals for three treatments by end of 2019
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[January 10, 2018] By
Bill Berkrot
(Reuters) - U.S. biotech bluebird bio Inc,
which does not yet have any marketed products, expects to be able to
file for regulatory approval of three novel treatments by the end of
2019, chief executive Nick Leschly announced on Tuesday.
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Bluebird expects to file for approval this year for its treatment
for transfusion-dependent beta thalassemia, which is intended to
eliminate the need for repeated transfusions for the inherited blood
disorder, Leschly said at the JP Morgan Healthcare Conference in San
Francisco.
The other two treatments are bluebird's CAR-T therapy for multiple
myeloma, being developed with partner Celgene Corp, and a treatment
for a rare and deadly genetic brain disorder, cerebral
adrenoleukodystrophy, both of which have demonstrated impressive
early results.

The company caused a stir at the American Society of Hematology
meeting in December with its CAR-T bb2121 as the therapy led to
extremely high response and remission rates with good safety in a
small study of heavily pretreated multiple myeloma patients who had
run out of options.
Leschly said the company expects to file for U.S. and European
approval of bb2121 in 2019.
Beyond those three, bluebird is also developing a promising
treatment for sickle cell disease, a potentially life-shortening
inherited blood disorder that causes severe pain and often leads to
repeat hospitalizations.
The company plans to discuss appropriate endpoints for future sickle
cell clinical trials with health regulators and will provide an
update on the program at the end of the year, the CEO said.
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He said the company is looking to develop "the right pricing and
reimbursement strategy" for treatments expected to be extremely
expensive. The first two approved CAR-T treatments for other cancers
from Novartis and Gilead Sciences were priced at $475,000 and
$373,000, respectively, for the one-time treatments.
As bluebird progresses toward a portfolio of potentially
game-changing commercial treatments, Leschly said one of his
overriding principles is: "Don't do stupid shortsighted stuff."
"We haven't done anything yet," Leschly said. "We need to deliver
products to patients."
(This version corrects name of multiple myeloma drug to bb2121 from
b2121 in fourth and fifth paragraphs)
(Reporting by Bill Berkrot; Editing by Tom Brown and Bill Trott)
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