r`F+LWo12>cc`*gZi{:.vC#?O$c-P y0c`~ G ^Y_ )s'4g Zynteglo (betibeglogene autotemcel) is an autologous hematopoietic stem cell-based gene therapy indicated for the treatment of adult and pediatric patients with beta-thalassemia who require regular red blood cell (RBC) transfusions. Treatment for: Beta Thalassemia This process takes approximately one week and may need to be repeated. packed red blood cell transfusions for a continuous period of 12 months at any time during the SOMERVILLE, Mass. Zynteglo FDA Approval History Last updated by Judith Stewart, BPharm on Aug 18, 2022. CHMP opinion/CMDh position date 4/13/2022 FDA PDUFA date for this cocktail . On 22 July 2021, . If you need a HIV test, talk with your healthcare provider about the appropriate test to use. You may receive more than one bag of ZYNTEGLO. There is a potential risk of blood cancer associated with this treatment; however, no cases have been seen in studies of ZYNTEGLO. endstream endobj startxref The therapy, previously known as beti-cel (betibeglogene autotemcel), will be marketed under the brand name Zynteglo, the same name for which it is sold in Europe. For adult and pediatric patients with beta-thalassemia who require regular red blood cell The FDA's clearances of Zynteglo and Skysona are a boost to Bluebird, and could help lift the research field after a series of setbacks. FDA Approved: Yes (First approved August 17, 2022) Brand name: Zynteglo Generic name: betibeglogene autotemcel Dosage form: Suspension for Intravenous Infusion Company: Bluebird Bio, Inc. It is important for you to be monitored at least yearly for at least 15 years for any changes to your blood. The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely. After billions of dollars of investment, the company's market valuation nosediving from nearly $10 billion in 2018 to just above $250 million currently and FDA delays, Zynteglo's approval is the much-needed vote of confidence . You will be admitted to the hospital for this step and remain in the hospital until after ZYNTEGLO infusion. The U.S. Food and Drug Administration approved bluebird bio Inc.'s cell-based gene therapy called Zynteglo. The FDA reported that 89% of the 41 patients taking Zynteglo in two clinical investigations, including adults and children with transfusion-dependent beta thalassemia, attained transfusion independence. This link will take you to a website that is outside the control of bluebird bio, Inc. We do not make or imply any endorsement of external websites. Stay up to date on the latest information about ZYNTEGLO. The most common side effects of ZYNTEGLO are: Call your healthcare provider right away if you have new or unusual bleeding which may include any of these signs or symptoms: You may experience side effects associated with other medicines administered as part of the ZYNTEGLO treatment regimen. Zynteglo is a one-time gene therapy product administered as a single dose. PRAC recommendation date : 09/07/2021. . Before it comes to the U.S. market, though, beti-cel must get the FDA's stamp of approval. The FDA approved betibeglogene autotemcel (Zynteglo; bluebird bio) for the treatment of adult and . Common Grade 3 or 4 laboratory abnormalities include neutropenia, thrombocytopenia, leukopenia, anemia, and lymphopenia. A small number of patients have been treated with Zynteglo to date, and t he most serious side effect observed is thrombocytopenia (low blood levels of . The information on this website does not take place of discussions with a healthcare professional and is not intended to provide medical advice. The biotech company set a $2.8 million price for the one-time . August 17, 2022 Approval Letter - ZYNTEGLO August 17, 2022 Summary Basis for Regulatory Action - ZYNTEGLO Approval History, Letters, Reviews and Related Documents - ZYNTEGLO Related. Call my bluebird support at 1-833-888-NEST (6378), The wholesale price of Bluebird is $2.8 million for the wholesale of the product, Zynteglo, which makes it among the most expensive therapies . study after ZYNTEGLO infusion. Altogether, these studies offer 220 patient-years worth of data and, as of March 9, span 63 pediatric, adolescent and adult patients of various genotypes who . Transfusion independence: a weighted average Hb 9 g/dL without any Lindsey Mulrooney. If you receive back-up cells, you will have no benefit from ZYNTEGLO. Why it matters: The one-time treatment which helps patients who depend on regular blood transfusions and face decreased life expectancy comes at a record price of $2.8 million. bluebird unveils $2.8m price for gene therapy Zynteglo on FDA approval Phil Taylor August 18, 2022 bluebird bio's Zynteglo has become the first cell-based gene therapy to be approved in. The most common side effects of ZYNTEGLO on the day of treatment are: Increased heart rate and Abdominal pain. Available for Android and iOS devices. . What should I avoid after receiving ZYNTEGLO? ZYNTEGLO (betibeglogene autotemcel) bluebird bio, Inc. ZOLGENSMA (onasemnogene abeparvovec-xioi) Novartis Gene . Generic name: betibeglogene autotemcel Approved name : Zynteglo. The agency also is responsible for the safety and security of our nations food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products. have read, understand, and agree to all the, agree for bluebird bio, Inc. and companies working on behalf of bluebird bio, Inc., to use the information provided on this form to stay in contact with you for marketing and educational purposes and/or provide information about products, and other activities from bluebird bio, Inc. including, but not limited to, market research, patient ambassador programs, and collecting patient and caregiver stories, Your privacy is important to us. August 17, 2022 To date, the FDA has not approved treatments for any form of EB. Shares are up 7% in Wednesday . The U.S. Food and Drug Administration (FDA) has approved ZYNTEGLO (betibeglogene autotemcel), a one-time gene therapy to treat patients with beta-thalassemia who require regular red blood cell (RBC) transfusions. U `Y This is a precaution in case there is a problem in the treatment process. US FDA approval tracker: June 2022. The most common adverse reactions associated with Zynteglo included reduced platelet and other blood cell levels, as well as mucositis, febrile neutropenia, vomiting, pyrexia (fever), alopecia (hair loss), epistaxis (nosebleed), abdominal pain, musculoskeletal pain, cough, headache, diarrhea, rash, constipation, nausea, decreased appetite, pigmentation disorder and pruritus (itch). ZYNTEGLO FDA APPROVAL AND COMMERCIAL LAUNCH PROGRESS. August 17, 2022. The most common side effects of ZYNTEGLO following treatment for up to 6 months are: Low level of platelets, which may reduce the ability of blood to clot and may cause bleeding, Low level of white blood cells, which may make you more susceptible to infection, and Pain in arms or legs. Following overwhelming support from an advisory committee, the FDA approved bluebird bio's gene therapy for beta-thalassemia on Wednesday.. CHMP endorses review finding no link between viral vector in Zynteglo and blood cancer. A month later, however, Bluebird said it . Zynteglo was approved for the treatment of adult and pediatric patients with beta-thalassemia who require regular red blood cell transfusions after safety and efficacy were demonstrated in clinical trials. The FDA has approved Zynteglo (betibeglogene autotemcel) for the treatment of patients with beta-thalassemia who require RBC transfusions. achieve transfusion independence.1, In two phase 3, open-label, single-arm studies, 89% (n=32/36; 95% CI: 74, 97) of patients* achieved transfusion independence.1. The safety and effectiveness of Zynteglo were established in two multicenter clinical studies that included adult and pediatric patients with beta-thalassemia requiring regular transfusions. You should consider discussing options for fertility preservation with your doctor before treatment. bluebird bio, the bluebird bio logo, ZYNTEGLO, and the ZYNTEGLO logo are trademarks of bluebird bio, Inc. (RBC) transfusions, ZYNTEGLO is a one-time gene therapy that gives patients the potential to "Our first product approval is a . To date, the current standard of care for people living with TDT involves transfusions up to every two to five . ZYNTEGLO is a one-time gene therapy to treat beta thalassemia (also known as beta thalassemia major or Cooleys Anemia) in patients who require regular transfusions. If you are a patient or caregiver and have questions about ZYNTEGLO, ask your healthcare professional. FDA adcoms also recommended that Covid vaccines from Moderna and . It is currently awaiting a PDUFA review, which is slated for the 19th of August, 2022. Dosage form: Suspension for Intravenous Infusion Data sources include IBM Watson Micromedex (updated 1 Nov 2022), Cerner Multum (updated 25 Oct 2022), ASHP (updated 12 Oct 2022) and others. Of those 41 patients, 36 were evaluable for transfusion independence. What is the most important information I should know about ZYNTEGLO? This website is developed by bluebird bio, Inc. and is intended for US residents 18 years of age and older only. STN: 125717 Why Issue Selected: Zynteglo (betibeglogene autotemcel), approved by the FDA in August of 2022, is the first cell-based gene therapy indicated for the treatment of adult and pediatric patients . Bluebird bio has suggested publicly that Zynteglo would be . The company behind the medicine, bluebird bio, has set Zynteglo's initial wholesale cost at $2.8 million, making it the most expensive medicine to ever reach the US market. The product is indicated for adult and pediatric patients who need regular red blood cell transfusions. The approval comes just days ahead of a number of important phase 3 read outs for Zynteglo at the European Hematology Association (EHA) congress in Amsterdam. Your healthcare provider will collect your blood stem cells though a process called mobilization and apheresis. Zynteglo is the third gene therapy approved by the FDA. Pending approval, along with positive data and the possibility to save patients money, may lead to a high cost for this gene therapy. SOMERVILLE, Mass.--(BUSINESS WIRE)-- bluebird bio Inc. (Nasdaq: BLUE) today announced the U.S. Food and Drug Administration (FDA) has approved ZYNTEGLO (betibeglogene autotemcel), also known as beti-cel, a one-time gene therapy custom-designed to treat the underlying genetic cause of betathalassemia in adult and pediatric patients who require regular red blood cell (RBC) transfusions. August 17, 2022 Today, the U.S. Food and Drug Administration approved Zynteglo (betibeglogene autotemcel), the first cell-based gene therapy for the treatment of adult and pediatric patients. hbbd```b``.WddcOhH&=0LZe/9``?0 13D$@ ;D: ?( PX U Your healthcare provider will determine when you can go home. Last updated by Judith Stewart, BPharm on June 1, 2022. Kymriah FDA Approval History. Additionally, we are readying SKYSONA for commercial availability and are on track to file our biologics licensing application for lovo-cel for sickle cell disease in Q1 2023. . The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. Given the potential health complications associated with this serious disease, this action highlights the FDAs continued commitment to supporting development of innovative therapies for patients who have limited treatment options.. This application was granted a rare pediatric disease voucher, in addition to receiving Priority Review, Fast Track, Breakthrough Therapy, and Orphan designations. The therapy is designed for the . What are additional possible or reasonably likely side effects of ZYNTEGLO? Action Due Date: . Talk to your physician regarding those possible side effects. Of 41 patients receiving Zynteglo, 89% achieved transfusion independence. There is a potential risk of blood cancer associated with this treatment; however, no cases have been seen in studies of Zynteglo. It was FDA panels galore last month, with good news for most. By Dark Horse Principal Kevin Whittlesey, Ph.D. Patients should also be monitored for hypersensitivity reactions during Zynteglo administration and should be monitored for thrombocytopenia and bleeding. Up next for approval are bluebird bio's Zynteglo lentiviral gene therapy and Immunocore's novel monoclonal T cell receptor therapy tebentafusp. Bluebird's medicine won't be cheap, though. Management team to host conference call Thursday, August 18 at 8:00 am ET. Coordinate the timing of Zynteglo thaw and ]f< - Treatment for: Beta Thalassemia. Sarah Silbiger/Getty Images. If you are diagnosed with a cancer, have your treating physician contact bluebird bio at 1-833-999-6378. Drugs.com provides accurate and independent information on more than 24,000 prescription drugs, over-the-counter medicines and natural products. On August 17, 2022, the U.S. Food and Drug Administration (FDA) approved ZYNTEGLO (betibeglogene autotemcel), the first one-time gene therapy custom-designed to treat the underlying genetic cause of betathalassemia in adult and pediatric patients who require regular red blood cell (RBC . Date: September 20, 2021 . ZYNTEGLO is for patients with beta-thalassemia who require regular RBC transfusions. The U.S. FDA has approved bluebird bio's ( NASDAQ: BLUE) Zynteglo (betibeglogene autotemcel), a gene therapy for the treatment of the blood disorder -thalassemia. Zynteglo's hefty price tag of $2.8 million is in line with it being a single, one-time administered gene therapy. FDA approval of Bluebird Bio's Zynteglo makes it the first gene therapy in the U.S. for the rare blood disorder beta thalassemia. International non-proprietary name (INN) or common name . The approval of Zynteglo is based on data from bluebird bio's phase 3 studies HGB-207 (Northstar-2) and HGB-212 (Northstar-3), and the long-term follow-up study LTF-303. SOMERVILLE, Mass., November 07, 2022--bluebird bio, Inc. (NASDAQ: BLUE) ("bluebird bio" or the "Company") today reported financial results and business highlights for the third quarter ended September 30, 2022 and shared recent operational progress. After ZYNTEGLO infusion, you will stay in the hospital for approximately 3-6 weeks so that your healthcare team can closely monitor your recovery. agree for bluebird bio, Inc. and companies working on behalf of bluebird bio, Inc., to use the information provided on this form to stay in contact with you for marketing and educational purposes and/or provide information about products, and other activities from bluebird bio, Inc. Low level of platelets, which may reduce the ability of blood to clot and may cause bleeding, Low level of white blood cells, which may make you more susceptible to infection. The reduced levels of red blood cells can lead to a number of health issues including dizziness, weakness, fatigue, bone abnormalities and more serious complications. On 3 June, Bluebird Bio Inc received approval from the European Medicines Agency (EMA) for its gene therapy Zynteglo (autologous CD34+ cells encoding A-T87Q-globin gene) for patients ages 12 years and over with . reserved. . For that reason, bluebird had decided voluntarily to suspend marketing of Zynteglo until the FDA's lifting of its clinical holds. Should it win approval in the U.S., initially Zynteglo would likely have a market of roughly 1,000 to 1,500 beta thalassemia patients. Key takeaways: In August 2022, the FDA approved Zynteglo (betibeglogene autotemcel) to treat severe beta-thalassemia, a genetic blood disorder, in kids and adults. %PDF-1.7 % Please see full Prescribing Information for ZYNTEGLO. %%EOF Kite Pharma, Inc. YESCARTA (axicabtagene ciloleucel) Kite Pharma, Incorporated. The .gov means its official.Federal government websites often end in .gov or .mil. ZYNTEGLO FDA APPROVAL AND COMMERCIAL LAUNCH PROGRESS. "The FDA approval of ZYNTEGLO offers people with beta-thalassemia the possibility of freedom from burdensome regular red blood cell transfusions and iron chelation, and unlocks new possibilities . Proposed Date: December 15, 2022 . Zynteglo is shipped to the treatment center in a frozen state and must be stored according to product specifications. . Proper Name: betibeglogene autotemcel CI = confidence interval. Bluebird gained recommendations for its gene therapies eli-cel and beti-cel; full approvals will be financially crucial as these would give the group priority review vouchers to sell. Atara on verge of landmark approval for first allogeneic T-cell therapy. The easiest way to lookup drug information, identify pills, check interactions and set up your own personal medication records. Patients with the most severe form of beta-thalassemia experience severe anemia and lifelong dependence on regular red blood cell transfusions, a lengthy process that patients typically undergo every 2-5 weeks. *The phase 3 studies evaluated the efficacy of ZYNTEGLO in 41 patients aged 4 to 34 years. ZYNTEGLO will not give you human immunodeficiency virus (HIV) infection. You are encouraged to report negative side effects of prescription drugs to the FDA. The site is secure. Beta-thalassemia is a rare, genetic blood disease caused by mutations in the beta-globin gene and characterized by significantly reduced or absent adult hemoglobin production. Subscribe to Drugs.com newsletters for the latest medication news, new drug approvals, alerts and updates. 431 0 obj <>stream MondayFriday, 8 AM8 PM ET to 14 June sees results from several trials, including the ongoing Phase 3 Northstar-3 (HGB-212) study in the most difficult to treat sub-group of patients who have 0 / 0 genotype or an . Beta-thalassemia is caused by a change in the beta-globin gene, which causes the body to produce reduced or no beta-globin. "Today's approval is an important advance in the treatment of beta-thalassemia, particularly in individuals who . Skysona, a treatment for an inherited neurological disorder called cerebral adrenoleukodystrophy, was approved by European regulators in July. Zynteglo is made specifically from a patient's own blood stem cells, which are collected by a process called mobilization and apheresis. Data from 41 . August 17, 2022. Klima said that Zynteglo will launch at a cost of $2.8 million per patient, adding that the . APPROVAL . Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances. Zynteglo's price of $1.8 million is higher than expected, with GlobalData anticipating a price of approximately $1.3-1.4 million. On August 17, 2022, the U.S. Food and Drug Administration (FDA) approved ZYNTEGLO(R) . Indication: An official website of the United States government, : with consideration of FDA comments. The U.S. Food and Drug Administration (FDA) approved N8-GP, or Esperoct, as a prophylactic (preventive) therapy to reduce frequency of bleeds and for on-demand use to control bleeding episodes in patients with inherited factor VIII deficiency, the company announced in a press release. Tradename: ZYNTEGLO coverage, and support for ZYNTEGLO. Each dose of Zynteglo is a customized treatment created using the patients own cells (bone marrow stem cells) that are genetically modified to produce functional beta-globin (a hemoglobin component). ZYN-US-00039 08/22. The approval of ZYNTEGLO is based on data from bluebird bio's Phase 3 studies HGB-207 (Northstar-2) and HGB-212 (Northstar-3), and the long-term follow-up study LTF-303. . Do not donate blood, organs, tissues or cells. Effectiveness was established based on achievement of transfusion independence, which is attained when the patient maintains a pre-determined level of hemoglobin without needing any red blood cell transfusions for at least 12 months. Manufacturer: bluebird bio Inc. The blood stem cells are then sent to a manufacturing site where they are used to make Zynteglo. Warnings and precautions associated with Zynteglo include delayed platelet engraftment, risk of neutrophil engraftment failure, risk of insertional oncogenesis, and hypersensitivity reactions. "The FDA approval of ZYNTEGLO offers people with beta-thalassemia the possibility of freedom from burdensome regular red blood cell transfusions and iron chelation, and unlocks new possibilities . The site is secure. Zynteglo (betibeglogene autotemcel) Consumer information. ZYNTEGLO FDA APPROVAL AND . We collect this information to help tailor the content that is delivered to you. After receiving the chemotherapy it may not be possible for you to become pregnant or father a child. Treatment with ZYNTEGLO may cause a false-positive HIV test result by some commercial tests. . Zynteglo represents the first ex-vivo lentiviral vector gene therapy to win FDA approval for beta-thalassemia. By Kathryn Hardison. August 17, 2022 - bluebird bio, Inc. announced the FDA has approved ZYNTEGLO (betibeglogene autotemcel), also known as beti-cel, a one-time gene therapy custom-designed to treat the underlying genetic cause of betathalassemia in adult and pediatric patients who require regular red blood cell (RBC) transfusions. Links are provided for informational purposes only. Aug. 17, 2022 at 2:13 p.m. Additionally, to date 12 payers have issued medical coverage policies that are favorably aligned to ZYNTEGLO's clinical evidence base. STEP 4: ZYNTEGLO is given by an intravenous infusion. This material is provided for educational purposes only and is not intended for medical advice, diagnosis or treatment. The cost of therapy is $2.8 million per treatment not including the cost of hospitalization. The FDA's approval of Zynteglo is based on data from bluebird's multiple Phase III clinical trials, which were part of the longest and most robust clinical gene therapy program in transfusion-dependent beta-thalassemia (TDT). Todays approval is an important advance in the treatment of beta-thalassemia, particularly in individuals who require ongoing red blood cell transfusions, said Peter Marks, M.D., Ph.D., director of the FDAs Center for Biologics Evaluation and Research. The Bluebird Bio gene therapy treats beta thalassemia, which is a rare blood disorder. ZYNTEGLO is made specifically for each patient, using the patients own blood stem cells and adds functional copies of the beta-globin gene to your cells. STEP 3: Before you receive ZYNTEGLO, your healthcare provider will give you chemotherapy for a few days to make room in the bone marrow. Vaccines, Blood & Biologics, Recalls, Market Withdrawals and Safety Alerts, Guidance, Compliance & Regulatory Information (Biologics), August 17, 2022 Approval Letter - ZYNTEGLO, August 17, 2022 Summary Basis for Regulatory Action - ZYNTEGLO, Approval History, Letters, Reviews and Related Documents - ZYNTEGLO, FDA Approves First Cell-Based Gene Therapy to Treat Adult and Pediatric Patients with Beta-thalassemia Who Require Regular Blood Transfusions, For treatment of adult and pediatric patients with -thalassemia who require regular red blood cell (RBC) transfusions, Demographic Subgroup Information betibeglogene autotemcel [ZYNTEGLO]. It is important that you have regular check-ups with your healthcare provider, including at least annual blood tests, to detect any adverse effects and to confirm that ZYNTEGLO is still working. SOMERVILLE, Mass., August 17, 2022--Following the FDA approval of ZYNTEGLO (betibeglogene autotemcel), also known as beti-cel, for the treatment of beta-thalassemia in adult and pediatric . (RBC) transfusions, ZYNTEGLOis a one-time gene therapy that gives patients the potential to achieve transfusion independence.1 In two phase 3, open-label, single-arm studies, 89% (n=32/36; 95% CI: 74, 97) of patients*achieved transfusion independence.1 Find a treatment center Learn about my bluebird support endstream endobj 395 0 obj <. Although Zynteglo is only administered one time, the treatment process involves several steps, including a lengthy hospitalization. Common non-laboratory adverse reactions include mucositis, febrile neutropenia, vomiting, pyrexia (fever), alopecia (hair loss), epistaxis (nose bleed), abdominal pain, musculoskeletal pain, cough, headache, diarrhea, rash, constipation, nausea, decreased appetite, pigmentation disorder, and pruritus (itch). Zynteglo is a one-time gene therapy used to treat beta . To date, the Company has signed outcomes-based agreements with pharmacy benefit managers (PBMs) representing more than 40 national and regional plans. Zynteglo is administered via intravenous infusion. locate a treatment center and learn more about educational resources, insurance The single-arm, open-label, 24-month phase 3 studies of Zynteglo included 41 patients aged 4 to 34 years with both non-0/0 and 0/0 genotypes, with longest follow up . STEP 2: Your blood stem cells will be sent to a manufacturing site where they are used to make ZYNTEGLO. Bluebird's application includes data from four clinical studies, two of which are Phase 3 trials. FDA Approved: Yes (First approved August 30, 2017) . Publish Date August 18, 2022 Today, the U.S. Food and Drug Administration approved Zynteglo (betibeglogene autotemcel), the first cell-based gene therapy for the treatment of adult and pediatric patients with beta-thalassemia who require regular red blood cell transfusions. The extension will allow the FDA to review additional clinical information that bluebird submitted in response to the regulator's requests. If this happens, your back-up stem cells will be given back to you. 0 . It takes approximately 70-90 days from the time your cells are collected to manufacture and test ZYNTEGLO before it is shipped to your healthcare provider, but the time may vary. Bluebird bio on Thursday said it will withdraw from market a rare disease gene therapy recently approved in Europe as the company winds down operations there. Procedure start date : 11/03/2021. The US Food and Drug Administration (FDA) has approved bluebird bio's Zynteglo (betibeglogene autotemcel; beti-cel), the first cell-based gene therapy for the treatment of adult and paediatric patients with beta-thalassemia who require regular red blood cell transfusions. Beta-thalassemia is a kind of inherited blood disorder that causes a reduction of normal hemoglobin and red blood cells (RBCs) in the blood through mutations in . This may allow you to produce sufficient hemoglobin to stop receiving regular transfusions. SKYSONA FDA APPROVAL AND COMMERCIAL LAUNCH PROGRESS Zynteglo helped about 89% of people avoid blood transfusions over a 12-month period in clinical trials. The forward-looking statements included in this document are made only as of the date of this document and except as otherwise required by applicable law, bluebird bio undertakes no obligation to . Created Date: 8/25/2022 11:32:33 AM . FDA Approves Zynteglo (betibeglogene autotemcel) Gene Therapy for People with Beta-Thalassemia Who Require Regular Red Blood Cell Transfusions. The FDA has approved Bluebird Bio's Zynteglo as the first cell-based gene therapy for the treatment of adult and pediatric patients with beta-thalassemia who require regular red . Call your doctor for medical advice about side effects. Date Article; Oct 1, 2021: Approval U.S. FDA Approves Kite's Tecartus as the First and Only CAR-T for Adults with Relapsed or Refractory B-Cell Acute Lymphoblastic Leukemia: Jul 24, 2020: Approval FDA Approves Tecartus (brexucabtagene autoleucel) as the First and Only CAR T Treatment for Relapsed or Refractory Mantle Cell Lymphoma: Feb 10, 2020: U.S. FDA Grants Priority Review for Kite's . August 17, 2022 Approval Letter - ZYNTEGLO August 17, 2022 Summary Basis for Regulatory Action - ZYNTEGLO Approval History, Letters, Reviews and Related Documents - ZYNTEGLO Related. 394 0 obj <> endobj ZYNTEGLO FDA APPROVAL AND COMMERCIAL LAUNCH PROGRESS. To date, Zynteglo has been the subject of a dozen clinical trials, including for sickle cell anemia. Each infusion bag is administered over a period of less than 30 minutes. Visit www.fda.gov/medwatch or call 1-800-FDA-1088. Before sharing sensitive information, make sure you're on a federal government site. We comply with the HONcode standard for trustworthy health information. 417 0 obj <>/Filter/FlateDecode/ID[<3FF73DBD0927E048AE20FCAE86CC96E4><29936B1AA95EF84ABB87232A8BE85621>]/Index[394 38]/Info 393 0 R/Length 105/Prev 263663/Root 395 0 R/Size 432/Type/XRef/W[1 3 1]>>stream Company: Bluebird Bio, Inc. These regular transfusions can be associated with multiple health complications of their own, including problems in the heart, liver and other organs due to an excessive build-up of iron in the body. This document summarizes the basis for regular approval of ZYNTEGLO. Esperoct, however, will not arrive on the U.S. market before . "Positive momentum continues to build through the ZYNTEGLO launch and we are on track to complete our first cell collection this quarter. The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely. The significance of the new approval is due to a variety of reasons, including that to date there are only a few FDA-approved gene Your healthcare providers will give you other medicines, including chemotherapy medicine, as part of your treatment with ZYNTEGLO. These are not all the possible side effects of ZYNTEGLO. 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