CAMBRIDGE, Mass.–(BUSINESS WIRE)–bluebird bio, Inc. (Nasdaq: BLUE) appear today that the European Medicines Agency (EMA) accustomed the company’s business allotment appliance (MAA) for its investigational LentiGlobin™ gene analysis for the analysis of adolescents and adults with transfusion-dependent β-thalassemia (TDT) and a non-β0/β0 genotype.
LentiGlobin was ahead accepted an accelerated appraisal by the Committee for Medicinal Products for Human Use (CHMP) of the EMA in July 2018, potentially abbreviation the EMA’s alive analysis time of the MAA from 210 canicule to 150 days.
“People active with transfusion-dependent β-thalassemia crave common claret transfusions that are life-saving but may advance to complications, including agency abortion due to adamant overload,” said David Davidson, M.D., arch medical officer, bluebird bio. “The accepting of our business allotment appliance for LentiGlobin is a anniversary that advances us against our ambition of accouterment to patients the aboriginal ancient gene analysis that addresses the basal abiogenetic account of TDT. We allotment this important anniversary with the patients, families and healthcare providers who fabricated it accessible through their accord in our beat analytic studies of LentiGlobin.”
The MAA for LentiGlobin is accurate by abstracts from the completed Phase 1/2 Northstar (HGB-204) abstraction and the advancing Phase 1/2 HGB-205 abstraction as able-bodied as accessible abstracts from the Phase 3 Northstar-2 (HGB-207) abstraction and the abiding aftereffect abstraction LTF-303.
About Transfusion-Dependent β-ThalassemiaTDT is an affiliated claret ataxia acquired by a alteration in the β-globin gene, which causes abortive red claret corpuscle assembly arch to astringent anemia. Admiring affliction for bodies with TDT consists of a constant dieting of abiding claret transfusions to accredit adaptation and aish affection of the disease, and adamant chelation analysis to administer adamant afflict that after-effects from the transfusions.
Despite the availability of admiring care, abounding bodies with TDT acquaintance austere complications and agency accident due to basal ache and adamant overload. By eliminating or abbreviation the charge for claret transfusions, the abiding complications associated with TDT may be reduced.
Allogeneic hematopoietic axis corpuscle transplantation (allo-HSCT) has been auspiciously acclimated to amusement TDT and is currently the alone accessible advantage with the abeyant to absolute the abiogenetic absence in TDT. Complications of allo-HSCT accommodate a accident of treatment-related mortality, affix failure, graft-versus-host ache (GvHD) and adept infections, decidedly in patients who abide non-sibling akin allo-HSCT.
About LentiGlobinLentiGlobin is a ancient gene analysis actuality advised as a abeyant analysis to abode the basal abiogenetic account of TDT, which could annihilate or abate the charge for claret transfusions.
bluebird bio’s analytic development affairs for LentiGlobin includes advancing studies about the apple with sites in Australia, Germany, Greece, France, Italy, Thailand, the United Kingdom and the United States. For added advice visit: www.northstarclinicalstudies.com or clinicaltrials.gov appliance identifier NCT01745120.
In addition, bluebird is administering a abiding assurance and ability aftereffect abstraction (LTF-303) for bodies who accept alternate in bluebird bio-sponsored analytic studies of LentiGlobin for TDT and sickle corpuscle disease.
The EMA ahead accepted Priority Medicines (PRIME) accommodation and Orphan Medicinal Artefact appellation to LentiGlobin for the analysis of TDT. LentiGlobin is additionally allotment of the EMA’s Adaptive Pathways pilot program, which is allotment of the EMA’s accomplishment to advance appropriate admission for patients to new medicines.
The U.S. Food and Drug Administration (FDA) additionally accepted LentiGlobin Orphan Drug cachet and Breakthrough Analysis appellation for the analysis of TDT.
About bluebird bio, Inc.With its lentiviral-based gene therapies, T corpuscle immunotherapy ability and gene alteration capabilities, bluebird bio has congenital a activity with ample abeyant appliance in astringent abiogenetic diseases and cancer.
bluebird bio’s gene analysis analytic programs accommodate investigational treatments for bookish adrenoleukodystrophy, transfusion-dependent β-thalassemia, additionally accepted as β-thalassemia major, and astringent sickle corpuscle disease.
bluebird bio’s oncology activity is congenital aloft the company’s lentiviral gene commitment and T corpuscle engineering, with a focus on developing atypical T cell-based immunotherapies, including chimeric antigen receptor (CAR T) and T corpuscle receptor (TCR) therapies. The company’s advance oncology programs are anti-BCMA CAR T programs partnered with Celgene.
bluebird bio’s analysis analysis programs accommodate utilizing megaTAL/homing endonuclease gene alteration technologies with the abeyant for use beyond the company’s pipeline.
bluebird bio has operations in Cambridge, Massachusetts; Seattle, Washington; Durham, North Carolina and Zug, Switzerland.
LentiGlobin is a brand of bluebird bio, Inc.
Forward-Looking StatementsThis absolution contains “forward-looking statements” aural the acceptation of the Private Securities Litigation Reform Act of 1995, including statements apropos the Company’s angle with account to the abeyant for its LentiGlobin artefact applicant to amusement transfusion-dependent ß-thalassemia, and the Company’s expectations apropos the review, abeyant authoritative approval and abeyant bartering barrage of its LentiGlobin artefact applicant in the United States and Europe. Any advanced statements are based on management’s accepted expectations of approaching contest and are accountable to a cardinal of risks and uncertainties that could account absolute after-effects to alter materially and abnormally from those set alternating in or adumbrated by such advanced statements. These risks and uncertainties include, but are not bound to, the risks that the basic absolute ability and assurance after-effects from our above-mentioned and advancing analytic trials of LentiGlobin will not abide or be again in our advancing or planned analytic trials of LentiGlobin, the risks that the changes we accept fabricated in the LentiGlobin accomplishment will not aftereffect in bigger accommodating outcomes, risks that the accepted or planned analytic trials of LentiGlobin will be bereft to abutment approaching authoritative submissions or to abutment business approval in the US and EU, and the accident that any one or added of our artefact candidates, will not be auspiciously developed, accustomed or commercialized. For a altercation of added risks and uncertainties, and added important factors, any of which could account our absolute after-effects to alter from those independent in the advanced statements, see the area advantaged “Risk Factors” in our best contempo Form 10-Q, as able-bodied as discussions of abeyant risks, uncertainties, and added important factors in our consecutive filings with the Securities and Exchange Commission. All advice in this columnist absolution is as of the date of the release, and bluebird bio undertakes no assignment to amend this advice unless appropriate by law.
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