bluebird bio Announces European Medicines Agency’s Acceptance of Marketing Authorization Application for LentiGlobin™ Gene Therapy for the Treatment of Transfusion-Dependent β-Thalassemia
bluebird bio, Inc. (Nasdaq: BLUE) announced today that the European Medicines Agency (EMA) accepted the company’s marketing authorization application (MAA) for its investigational LentiGlobin™ gene therapy for the treatment of adolescents and adults with transfusion-dependent β-thalassemia (TDT) and a non-β0/β0 genotype.
LentiGlobin was previously granted an accelerated assessment by the Committee for Medicinal Products for Human Use (CHMP) of the EMA in July 2018, potentially reducing the EMA’s active review time of the MAA from 210 days to 150 days.
“People living with transfusion-dependent β-thalassemia require frequent blood transfusions that are life-saving but may lead to complications, including organ failure due to iron overload,” said David Davidson, M.D., chief medical officer, bluebird bio. “The acceptance of our marketing authorization application for LentiGlobin is a milestone that advances us toward our goal of providing to patients the first one-time gene therapy that addresses the underlying genetic cause of TDT. We share this important milestone with the patients, families and healthcare providers who made it possible through their participation in our pioneering clinical studies of LentiGlobin.”
The MAA for LentiGlobin is supported by data from the completed Phase 1/2 Northstar (HGB-204) study and the ongoing Phase 1/2 HGB-205 study as well as available data from the Phase 3 Northstar-2 (HGB-207) study and the long-term follow-up study LTF-303.
About Transfusion-Dependent β-Thalassemia
TDT is an inherited blood disorder caused by a mutation in the β-globin gene, which causes ineffective red blood cell production leading to severe anemia. Supportive care for people with TDT consists of a lifelong regimen of chronic blood transfusions to enable survival and suppress symptoms of the disease, and iron chelation therapy to manage iron overload that results from the transfusions.
Despite the availability of supportive care, many people with TDT experience serious complications and organ damage due to underlying disease and iron overload. By eliminating or reducing the need for blood transfusions, the long-term complications associated with TDT may be reduced.
Allogeneic hematopoietic stem cell transplantation (allo-HSCT) has been successfully used to treat TDT and is currently the only available option with the potential to correct the genetic deficiency in TDT. Complications of allo-HSCT include a risk of treatment-related mortality, graft failure, graft-versus-host disease (GvHD) and opportunistic infections, particularly in patients who undergo non-sibling matched allo-HSCT.
LentiGlobin is a one-time gene therapy being studied as a potential treatment to address the underlying genetic cause of TDT, which could eliminate or reduce the need for blood transfusions.
bluebird bio’s clinical development program for LentiGlobin includes ongoing studies around the world with sites in Australia, Germany, Greece, France, Italy, Thailand, the United Kingdom and the United States. For more information visit: www.northstarclinicalstudies.com or clinicaltrials.gov using identifier NCT01745120.
In addition, bluebird is conducting a long-term safety and efficacy follow-up study (LTF-303) for people who have participated in bluebird bio-sponsored clinical studies of LentiGlobin for TDT and sickle cell disease.
The EMA previously granted Priority Medicines (PRIME) eligibility and Orphan Medicinal Product designation to LentiGlobin for the treatment of TDT. LentiGlobin is also part of the EMA’s Adaptive Pathways pilot program, which is part of the EMA’s effort to improve timely access for patients to new medicines.
The U.S. Food and Drug Administration (FDA) also granted LentiGlobin Orphan Drug status and Breakthrough Therapy designation for the treatment of TDT.
About bluebird bio, Inc.
With its lentiviral-based gene therapies, T cell immunotherapy expertise and gene editing capabilities, bluebird bio has built a pipeline with broad potential application in severe genetic diseases and cancer.
bluebird bio's gene therapy clinical programs include investigational treatments for cerebral adrenoleukodystrophy, transfusion-dependent β-thalassemia, also known as β-thalassemia major, and severe sickle cell disease.
bluebird bio's oncology pipeline is built upon the company's lentiviral gene delivery and T cell engineering, with a focus on developing novel T cell-based immunotherapies, including chimeric antigen receptor (CAR T) and T cell receptor (TCR) therapies. The company’s lead oncology programs are anti-BCMA CAR T programs partnered with Celgene.
bluebird bio’s discovery research programs include utilizing megaTAL/homing endonuclease gene editing technologies with the potential for use across the company's pipeline.
bluebird bio has operations in Cambridge, Massachusetts; Seattle, Washington; Durham, North Carolina and Zug, Switzerland.
LentiGlobin is a trademark of bluebird bio, Inc.
This release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the Company’s views with respect to the potential for its LentiGlobin product candidate to treat transfusion-dependent ß-thalassemia, and the Company’s expectations regarding the review, potential regulatory approval and potential commercial launch of its LentiGlobin product candidate in the United States and Europe. Any forward-looking statements are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risks that the preliminary positive efficacy and safety results from our prior and ongoing clinical trials of LentiGlobin will not continue or be repeated in our ongoing or planned clinical trials of LentiGlobin, the risks that the changes we have made in the LentiGlobin manufacturing will not result in improved patient outcomes, risks that the current or planned clinical trials of LentiGlobin will be insufficient to support future regulatory submissions or to support marketing approval in the US and EU, and the risk that any one or more of our product candidates, will not be successfully developed, approved or commercialized. For a discussion of other risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the section entitled “Risk Factors” in our most recent Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in our subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and bluebird bio undertakes no duty to update this information unless required by law.
Om Business Wire
(c) 2018 Business Wire, Inc., All rights reserved.
Business Wire, a Berkshire Hathaway company, is the global leader in multiplatform press release distribution.
Følg saker fra Business Wire
Registrer deg med din epostadresse under for å få de nyeste sakene fra Business Wire på epost fortløpende. Du kan melde deg av når som helst.
Siste saker fra Business Wire
IFF Recognized by SAM’s 2019 “Industry Mover” Sustainability Award15.2.2019 21:15:00 | Pressemelding
Regulatory News: International Flavors & Fragrances Inc. (NYSE: IFF) (Euronext Paris: IFF) (TASE: IFF), was recognized for its sustainability efforts with the 2019 “Industry Mover” award from SAM, a subsidiary of RobecoSAM which specializes in providing environmental, social and governance (ESG) data, benchmarks and ratings. The recognition acknowledges the Company’s top-scoring performance in economic, social and environmental categories. “We are thrilled to receive this award from SAM,” said Andreas Fibig, IFF Chairman & CEO. “Our sustainability efforts are not only a key enabler of our overall strategy, but also a true passion here at IFF -- and we place it at the forefront of everything we do. It is deeply gratifying to have our work and our people recognized for doing the right thing for the planet.” SAM’s 2019 scoring is based on its 2018 Corporate Sustainability Assessment. Using a weighted methodology, SAM evaluates companies for criteria such as: codes of business conduct, ope
SFL: strong financial position and results in 2018, consolidating the Group’s position as a prime player15.2.2019 18:01:00 | Pressemelding
Regulatory News: The financial statements for the year ended 31 December 2018 were approved by the Board of Directors of Société Foncière Lyonnaise (Paris:FLY) on 15 February 2019 at a meeting chaired by Juan José Brugera. 2018 business indicators were very robust, with further underlying growth in rental income and historically high EPRA earnings. The portfolio's appraisal value and the Company's net asset value also continued to grow, attesting to SFL’s excellent positioning. The auditors have completed their audit of the annual financial information and are in the process of issuing their report. Consolidated data (€ millions) 2018 2017 Change Rental income 193.5 195.8 -1.2% Adjusted operating profit* 162.1 164.1 -1.2% Attributable net profit 351.6 685.3 - EPRA earnings 106.7 102.4 +4.1% * Operating profit before disposal gains and losses and fair value adjustments 31/12/2018 31/12/2017 Change Attributable equity 4,010 3,763 +6.6% Consolidated portfolio value excluding transfer cost
5 Happening Cultural Destinations to Visit in 201915.2.2019 10:11:00 | Pressemelding
Hong Kong There’s never really a sleepy year to visit Hong Kong, but 2019 is particularly ripe for visitors. Spearheaded by the dynamic entrepreneur Adrian Cheng of K11 and New World Development, the rejuvenation of Tsim Sha Tsui harbourfront is just the starting point. The city’s iconic harbourside walkway, Avenue of Stars, has just received a much-awaited makeover that’s the brainchild of Cheng and visually conceived by James Corner, the landscape architect responsible for the High Line in New York. It sees the addition of design-driven rest areas, kiosks featuring homegrown brands, hand prints from Asian film stars and interactive digital elements. Featuring Hong Kong’s first wave energy demonstrator that produces electricity for the Avenue, the place is turned into one of the most sustainable promenades in Hong Kong. This is just one part of Cheng and his company’s grander urban revitalisation plan, Victoria Dockside, a new art and design district which will fully open in the third
ISAE-SUPAERO Launches a New MOOC about Flight Mechanics15.2.2019 09:36:00 | Pressemelding
"ABC of Flight Mechanics: How Do Planes Fly?": a MOOC dedicated to flight mechanics. The notion of flight often defies our human terrestrial instincts. How does an airplane fly? How can a pilot maintain control? How high in the sky can an aircraft climb? How does a fighter pilot fly while spinning? This online course (available in English) will cover many topics, such as atmospheric physics and Newton's laws applied to aircraft, as well as propulsion forces and longitudinal stability the question of traction. The MOOC "ABC of Flight Mechanics" is aimed at general public with a specific level of scientific knowledge. Nevertheless, various levels of learning are available, thus it allows all students to take the course without needing to plenty understand the mathematical principles. Undergraduate students in aerospace engineering, trainees as well as novice and experienced pilots, journalists and professionals in the aeronautics domain (business, engineering, marketing, communications,
REPLY: Red Reply Wins the Oracle “Partner of the Year Autonomous” Award15.2.2019 09:30:00 | Pressemelding
Red Reply, the Reply group company that specialises in the Oracle Cloud IaaS and PaaS platform has been named as Oracle “Partner of the Year: Autonomous.” The award recognises Red Reply for its work as an Oracle Cloud Managed Service Provider Partner in developing highly innovative projects using the Oracle Autonomous Database technology. Red Reply has launched a project aimed at re-engineering the Policy Enterprise DataWarehouse of Verti Assicurazioni, company operating in the online insurance sector, using the Oracle Cloud Autonomous DataWarehouse service. The Autonomous DataWarehouse is a cloud-based database that uses machine learning to eliminate manual labour in operations such as tuning, security, backups, updates and other routine activities. This selected Oracle solution is the only one of its kind capable of addressing aspects such as security, performance and high availability of data, using automatic update and tuning mechanisms, while maximising the protection of data in l
Posiflex showcases new Interactive Self-Service Kiosks and IoT Retail Product Innovations at EuroCIS 201915.2.2019 08:00:00 | Pressemelding
The Posiflex Group — a synergy of world-leading POS, kiosk, and industrial computing technologies — will introduce its full product portfolio and latest innovations at EuroCIS, the leading trade fair for retail technology, from February 19th – 21st in Düsseldorf. With a product strategy focus on serviced IoT, the Posiflex Group will be displaying smart and connected retail solutions with fully-integrated middleware, RMS (Remote Monitoring System) and hardware. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20190215005008/en/ Posiflex at EuroCIS Hall 9 / C42 (Graphic: Business Wire) “I am excited to introduce the Posiflex Group portfolio to the European market,” says Hans Peter Nüdling, the newly appointed Chief Strategy Officer for the Posiflex Group. “The Posiflex Group references the collective brands of the corporation, including Posiflex, Portwell and KIOSK Information Systems (KIOSK) – all under one corporate umbrella. As