CAMBRIDGE, Mass.–(BUSINESS WIRE)–Vandaag is een persbericht van een derde partij uitgegaan waarin staat dat het EMA (European Medicines Agency) de goedkeuring heeft verleend voor de voorwaardelijke Marketing Authorisation Application (MAA) voor LentiGlobin™, bluebird bio’s onderzoekende gentherapie voor de behandeling van transfusie-afhankelijke β-thalassemie (TDT). LentiGlobin voor TDT zal naar verwachting worden bekeken als onderdeel van de vergadering van het CHMP (Committee on Human Medicinal Products) van 25 – 28 maart, maar het CHMP heeft geen advies uitgebracht. Als het advies van het CHMP wordt uitgebracht, wordt het beoordeeld door de Europese Commissie (EC), die bevoegd is om goedkeuring te verlenen voor het gebruik van LentiGlobin in de EU.
bluebird bio Statement on European Regulatory Status of LentiGlobin™
CAMBRIDGE, Mass.–(BUSINESS WIRE)– A third party press release was issued today stating that the EMA (European Medicines Agency) issued an approval for the conditional Marketing Authorization Application (MAA) for LentiGlobin™, bluebird bio’s investigational gene therapy for the treatment of transfusion dependent β-thalassemia (TDT). LentiGlobin for TDT is scheduled to be reviewed as part of the CHMP (Committee on Human Medicinal Products) meeting from March 25 – 28, however no opinion has been issued by the CHMP. If the CHMP’s opinion is issued it would then be reviewed by the European Commission (EC), which has the authority to grant approval for the use of LentiGlobin in the EU.
About bluebird bio, Inc.
bluebird bio is pioneering gene therapy with purpose. From our Cambridge, Mass., headquarters, we’re developing gene therapies for severe genetic diseases and cancer, with the goal that people facing potentially fatal conditions with limited treatment options can live their lives fully. Beyond our labs, we’re working to positively disrupt the healthcare system to create access, transparency and education so that gene therapy can become available to all those who can benefit.
bluebird bio is a human company powered by human stories. We’re putting our care and expertise to work across a spectrum of disorders by researching cerebral adrenoleukodystrophy, sickle cell disease, transfusion-dependent β-thalassemia and multiple myeloma using three gene therapy technologies: gene addition, cell therapy and (megaTAL-enabled) gene editing.
bluebird bio has additional nests in Seattle, Wash.; Durham, N.C.; and Zug, Switzerland. For more information, visit bluebirdbio.com.
This release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the Company’s plans and expectations for the development, regulatory approval, and commercialization for the LentiGlobin™ in TDT to treat transfusion-dependent β-thalassemia, and the potential implications of clinical data for patients. 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 risk that our MAA submitted for LentiGlobin may not be approved by the European Commission when expected, or at all; the risk that the 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 risk that the current or planned clinical trials of LentiGlobin will be insufficient to support regulatory submissions or marketing approval in the US and EU; the risk that the production of HbAT87Q may not be sustained over extended periods of time; and the risk that we may not secure adequate pricing or reimbursement to support continued development or commercialization of LentiGlobin following regulatory approval. 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-K, 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.