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
Transphorm Strengthens 900 V GaN Portfolio with Second FET25.6.2019 13:00:00 CEST | Pressemelding
Transphorm Inc.—the leader in the design and manufacturing of the highest reliability high-voltage (HV) Gallium Nitride (GaN) semiconductors—today introduced its second 900 V FET, the Gen III TP90H050WS, enhancing the industry’s only 900 V GaN product line. These devices now enable three-phase industrial systems and higher voltage automotive electronics to leverage GaN’s speed, efficiency and power density. Further, the new FET’s platform is based on Transphorm’s 650 V predecessor, the only JEDEC- and AEC-Q101-qualified HV GaN technology. As such, system developers can design with confidence in its quality and reliability. The TP90H050WS has a typical on-resistance of 50 mOhm with a 1000 V transient rating, offered in a standard TO-247 package. The TP90H050WS can reach power levels of 8 kW in a typical half bridge while maintaining greater than 99 percent efficiencies. Its figures of merit for Ron*Qoss (resonant switching topologies) and Ron*Qrr (hard switching bridge topologies) are t
Modern Governance 3.0: Modern Governance Extends Beyond the Boardroom to Allow Secure Sharing of Files and Collaboration with Auditors, Regulators and Other Third Parties25.6.2019 13:00:00 CEST | Pressemelding
In its next major extension to support modern governance, Diligent announced today the launch of Secure File Sharing and Secure Meeting Workflow applications, as part of the Diligent Governance Cloud. These groundbreaking applications enable completely secure collaboration not only between board members and executives, but also reaching beyond the boardroom including auditors, regulators and other key third party stakeholders. This significantly raises the bar on security and information governance for organizations operating in this digital, mobile and social era. In today’s fast-paced world, threats come at light speed – from every angle. Leading organizations must set increasingly higher standards to protect against cyber attacks and leaks, while at the same time work collaboratively with more outside parties. Even competent leaders can have blind spots when it comes to the handling of highly sensitive files, specifically in preparation for a board meeting, sharing documents haphaza
Moody’s Analytics CreditLens™ Solution Adopted by Over 100 Firms25.6.2019 13:00:00 CEST | Pressemelding
Moody’s Analytics, a leading provider of financial intelligence, announced today that the CreditLens credit-lifecycle-management solution has been adopted by over 100 firms since its launch. Built on the latest cloud-based technology, the CreditLens platform helps businesses digitally transform their commercial credit processes to make faster and better-informed decisions. The platform uses the latest AI and machine learning technology to facilitate process automation, and help clients improve efficiency, reduce errors, and streamline workflows. "We are honored that our clients have embraced the CreditLens solution and enabled us to reach this milestone,” said Annie Choi, Senior Director at Moody’s Analytics. “Our goal is to provide the best credit lifecycle management solution in the market, informed by our award-winning proprietary data, and enabled by the modern technologies our clients want to use.” “Technological innovation is accelerating at a rapid pace, creating opportunities f
BOC Group Announces the Release of ADONIS NP 7.1 – Igniting A New Era of User Inclusion, Insights & BPM Experience25.6.2019 12:26:00 CEST | Pressemelding
Today, BOC Group released the latest version of its revolutionary BPM suite, ADONIS NP 7.1. Focusing specially on enriching the ADONIS NP experience for every user, transparency, simplicity and openness were kept at the core of this version’s design. And this release’s flagship feature, the Process Insights Dashboard, is a direct embodiment of that, with its powerful visualization and representation of all key process management information. Tobias Rausch, ADONIS NP product manager, elaborates: “We carefully designed the Insights Dashboard in a way that provides that one-click-access to everything you would need to know about your processes – from process hierarchy, responsible roles, inputs, outputs, to IT, risks, KPIs, as well as quick-links to analyses and reports”. “Essentially, the Insights Dashboard brings an entirely new and simplified way of interacting with the process content, ultimately making ADONIS NP even more user-friendly and intuitive than befo re!" What’s more, the la
MATRIXX Software Joins Linux Foundation Networking to Advance Next Generation of Telco Services25.6.2019 12:05:00 CEST | Pressemelding
MATRIXX Software , an innovation powerhouse committed to transforming global commerce, today announced it has joined Linux Foundation Networking (LFN) as a silver member. MATRIXX is participating in the foundation’s programs to provide guidance related to advancing a new generation of services inspired by web-scale best practices. With over 100 members, LFN continues its global drumbeat of ecosystem growth for accelerated development and adoption of open source and open standards-based networking technologies. The Linux Foundation supports the creation of sustainable open source ecosystems by providing financial and intellectual resources, infrastructure, services, events and training. “The LF Networking community is working together to foster industry collaboration and innovation across the entire open networking stack, and increase harmonization across platforms, ecosystems and communities,” said Arpit Joshipura, general manager, Networking, Automation, Edge & IOT, the Linux Foundati
Patient Safety Movement Foundation Announces First Curriculum Commitments25.6.2019 12:00:00 CEST | Pressemelding
To advance the education of healthcare professionals and reduce the number of preventable deaths, the Patient Safety Movement Foundation (PSMF) has developed core curriculum to teach patient safety throughout the entire education process, from freshman year through residency. The continued education and development of healthcare students and professionals improves the quality of patient care and eliminates preventable medical error. By committing to the Patient Safety Curriculum (Actionable Patient Safety Solutions #17), participants commit to: Improve knowledge of the science, as well as the human side, of patient safety Gain skills required to apply safe care practices to reduce medical error Value the role of patients and families as members of the healthcare team Demonstrate effective personal and team behaviors that support a just culture and reliable health system performance Advance the goal of eliminating preventable patient deaths “Helping healthcare students and professionals