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.
For more than 50 years, Business Wire has been the global leader in press release distribution and regulatory disclosure.
Tilaa tiedotteet sähköpostiisi
Haluatko tietää asioista ensimmäisten joukossa? Kun tilaat mediatiedotteemme, saat ne sähköpostiisi välittömästi julkaisuhetkellä. Tilauksen voit halutessasi perua milloin tahansa.
Lue lisää julkaisijalta Business Wire
Denodo Offers Data Virtualization Test Drive on Microsoft Azure17.12.2018 11:30 | Tiedote
Denodo, the leader in data virtualization today announced the availability of a free Test Drive environment on Microsoft Azure. Following on the heels of its recently announced Test Drive on AWS, the newest Denodo Test Drive empowers users to quickly and easily create real-time data delivery without replication and explore the benefits of using the Denodo Platform on Azure. Denodo Test Drives are completely free of charge for demonstration, education and evaluation purposes and are available at: http://bit.ly/2AQddE3 Built on Azure, the Test Drive lets individuals discover, explore, prepare, and access data in a user-friendly interface for true self-service. The Denodo Test Drive solves some of the most pressing challenges faced by data scientists and data analysts by enabling them to integrate large volumes of data to support their agile BI, big data analytics and Web/cloud integration objectives. “Microsoft’s commitment to offer an ecosystem of cloud solutions makes it ideal for Deno
Arthur D. Little Report Outlines €200 Billion Digital Transformation Opportunity for Telecoms Sector17.12.2018 11:30 | Tiedote
Arthur D. Little (ADL) today published a new research report highlighting the need for greater digitalization in the global telecoms industry. The TIME 2018 Flagship Report outlines the scale of the digital opportunity, with analysis estimating that a ‘digital dividend’ of over €200 billion free cash flow awaits telcos that can successfully transform. Industry executives surveyed for the report confirm the findings. 73% say that digital will add value to their businesses, with key benefits including greater agility, increased subscriber growth and cost savings. CAPEX spending on areas such as 5G infrastructure and the Internet of Things is expected to require a 7% CAGR increase from 2017-2022, outpacing forecast revenue growth. Consequently, failing to digitally transform risks commoditization and disintermediation, leaving telcos unable to invest sufficiently. As one senior executive put it bluntly, “Digitalization – there is no other choice if we want to survive.” The report highligh
Arthur D. Little Global Automotive Study Outlines Importance of Car in Changing World of Mobility17.12.2018 11:30 | Tiedote
Arthur D. Little (ADL) today released its worldwide automotive market report. Key findings are that desire for car ownership remains strong, especially amongst the young, and that drivers are considering a dramatic switch to electric and hybrid vehicles (EVs). This will impact manufacturer and supplier engine strategies and push for faster development of EV ecosystems, such as charging solutions. Drivers are now much more willing to embrace alternative drivetrains. 58% would accept paying more for hybrids, and 50% for EVs. While the majority have petrol or diesel-engined vehicles, this could change rapidly. Of those planning to replace their car, 48% said they’d consider an EV (BEV, hybrid or plugin hybrid), 36% a petrol-engined vehicle and 12% diesel. Klaus Schmitz, Partner with Arthur D. Little explains: “Extending our groundbreaking 2015 automotive study shows enormous changes. While attachment to car ownership remains high, meaning the overall automotive market will remain stable,
Montagu Evans Chooses MobileIron Cloud to Keep Client Data Safe on Mobile Devices17.12.2018 11:00 | Tiedote
MobileIron (NASDAQ:MOBL), the secure foundation for modern work, today announced that Montagu Evans has selected MobileIron unified endpoint management (UEM) cloud solution, including MobileIron Access and MobileIron Threat Defense. For nearly a century, Montagu Evans, a partnership and one of UK’s largest owner-managed surveying firms, has helped its clients shape some of UK’s largest commercial, residential and mixed-use property development projects. With increasingly sensitive client work, more employees on job sites using mobile devices, and the newly enforced GDPR, it was imperative for Montagu Evans to use a best-in-class UEM solution to securely manage all data without compromising productivity. Safety from Mobile Threats Since Montagu Evans’ employees often need to connect through unsecured networks, such as public Wi-Fi networks, the company needed to ensure that highly sensitive customer information could not be intercepted or compromised. To solve this problem, Montagu Evan
Safebridge Takes Maritime Certification into The Digital Era with myCert17.12.2018 11:00 | Tiedote
Safebridge expands its product portfolio with the launch of myCert, an innovative digital platform which utilises the blockchain technology to provide a secure online environment and upgrade the entire maritime certification ecosystem. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20181217005042/en/ myCert – the first digital solution for maritime certificates (Photo: Safebridge) myCert connects three main components: the entities who issue certificates, the seafarers who hold and need those certificates in their everyday activity and the verifiers who can be virtually anyone wishing to verify the validity of the certificates. myCert is the solution for professional users to better manage the life cycle of certificates, by having control over them from one single platform, securely and with 24/7 availability worldwide. “We saw Safebridge’s myCert in action at INTERTANKO’s Seafarers’ Vetting Seminar, in the Philippines. Over 1
Price f(x) Files Petitions with US Patent and Trademark Office to Invalidate Vendavo’s Patents and Launches a Counter Lawsuit17.12.2018 10:08 | Tiedote
Price f(x), the global leader in cloud pricing software, has filed petitions for “Covered Business Method (“CBM”) Review” of four Vendavo patents, and will imminently file a fifth, which together will cover challenges to all of the patent claims that Vendavo has asserted in litigation between the parties. In Price f(x)’s view, each of Vendavo’s patents qualifies for the specialized CBM procedure created by US Congress to “crackdown on low-quality business method patents.” Price f(x)’s petitions challenge Vendavo’s patents on a variety of grounds, including in all cases that the claims are directed to patent-ineligible abstract ideas (e.g., math for pricing of products), and in many cases that Vendavo simply tried to claim more than it actually invented. The petitions are now pending in the U.S. Patent Office, which should make an initial ruling on Price f(x)’s petitions in approximately six months. Price f(x) is concurrently moving to stay the patent portion of the parties’ litigation
Uutishuoneessa voit lukea tiedotteitamme ja muuta julkaisemaamme materiaalia. Löydät sieltä niin yhteyshenkilöidemme tiedot kuin vapaasti julkaistavissa olevia kuvia ja videoita. Uutishuoneessa voit nähdä myös sosiaalisen median sisältöjä. Kaikki STT Infossa julkaistu materiaali on vapaasti median käytettävissä.Tutustu uutishuoneeseemme