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<\/a>\n\u201cThe data presented at EHA and published in NEJM underscore the consistent, durable and transformative benefits CASGEVY can provide to people living with sickle cell disease or transfusion-dependent beta thalassemia from early in life,\u201d said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex.<\/p>
\n\u201cDespite optimized supportive therapy, children living with sickle cell disease and transfusion\u2011dependent beta thalassemia carry a significant disease burden from a very young age, with progressive complications leading to the irreversible and life-shortening consequences of these diseases,\u201d said Franco Locatelli, M.D., Ph.D., Professor of Pediatrics at the Catholic University of the Sacred Heart of Rome, Director of the Department of Pediatric Hematology and Oncology at Bambino Ges\u00f9 Children\u2019s Hospital, Chair of Vertex\u2019s TDT Program Steering Committee, and presenting author of the 5\u201311 years old CASGEVY data at EHA. \u201cThese data represent a profoundly important step forward, and I look forward to the possibility of providing earlier intervention to prevent complications in children and for families who have had limited potentially curative options to date.\u201d<\/p>
\nCASGEVY clinical data for children ages 5\u201311 presented at EHA and published in NEJM<\/span><\/b><\/p> \nData from an interim analysis of the CLIMB-151 and CLIMB-141 studies highlight the transformative potential CASGEVY can provide to children ages 5\u201311 and are consistent with the durable clinical profile established in adult and adolescent patients. Collectively, these findings highlight the potential benefits of addressing vaso-occlusive crises (VOCs) and transfusion burden earlier in life, which can begin in childhood and are associated with cumulative, long-term complications in SCD and TDT including organ damage.<\/p> \nGlobal regulatory submissions to expand use of CASGEVY<\/span><\/b><\/p> \nCASGEVY is currently approved for eligible people 12 years and older with SCD with recurrent VOCs or TDT in several countries around the world. In the United States the regulatory review is underway with the FDA to expand the use of CASGEVY to younger children after Vertex was awarded the Commissioner\u2019s National Priority Voucher. Vertex has also recently completed regulatory submissions in the Kingdom of Saudi Arabia and United Kingdom to expand the use of CASGEVY to younger children. Upon availability, there is an established network of activated authorized treatment centers in these countries prepared to support patients.<\/p> \nThe use of CASGEVY in children ages 5\u201311 years is investigational.<\/p> \nAbout Sickle Cell Disease (SCD)<\/b><\/p> \nSickle Cell Disease (SCD) is a rare serious, inherited blood disease that is progressive and life\u2011shortening. The disease causes red blood cells to become rigid and misshapen, restricting blood flow and oxygen delivery to vital organs. Recurrent vaso\u2011occlusive crises (VOCs), unpredictable episodes of severe pain caused by blocked blood vessels, are a defining feature of SCD and frequently require hospitalization. Many patients experience these complications early in life, and over time, repeated VOCs and chronic anemia lead to progressive and irreversible organ damage, including damage to the brain, lungs, kidneys and heart. SCD places a substantial burden on patients and their families, who must manage frequent medical visits, hospitalizations, school and work disruptions, and the emotional toll of chronic pain and life\u2011threatening complications. Despite lifelong treatment, people with SCD and recurrent VOCs in Europe face shortened life expectancy, with a mean age of death of around 40 years, and report quality\u2011of\u2011life outcomes far below the general population.<\/p> \nAbout Transfusion\u2011Dependent Beta Thalassemia (TDT)<\/b><\/p> \nTransfusion\u2011dependent beta thalassemia (TDT) is a rare serious, inherited blood disease that is progressive and life\u2011shortening. The disease impairs the body\u2019s ability to produce sufficient hemoglobin, limiting oxygen delivery to tissues and organs. People with TDT do not have enough functional hemoglobin in their red blood cells and require regular, lifelong blood transfusions, often beginning early in childhood, along with ongoing iron chelation therapy. While transfusions are necessary for survival, many of the long\u2011term complications of TDT are exacerbated by chronic transfusion therapy and iron overload and cumulative damage to the heart, liver and endocrine system, as well as bone abnormalities and delayed growth and puberty. TDT places a significant and ongoing burden on patients and their families, requiring frequent medical visits and complex lifelong treatment. Despite lifelong treatment, people with TDT face shortened life expectancy, with a mean age of death of approximately 50\u201355 years in Europe, reduced quality of life and productivity, and significant use of health care resources.<\/p> \nAbout CASGEVY\u00ae<\/sup> (exagamglogene autotemcel)<\/strong><\/p> \nCASGEVY is a non-viral, ex vivo<\/i> CRISPR\/Cas9 gene-edited cell therapy for eligible patients with SCD or TDT, in which a patient\u2019s own hematopoietic stem and progenitor cells are edited at the erythroid specific enhancer region of the BCL11A<\/i> gene through a precise double-strand break. This edit results in the production of high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is the form of the oxygen-carrying hemoglobin that is naturally present during fetal development, which then switches to the adult form of hemoglobin after birth. CASGEVY has been shown in clinical trials to reduce or eliminate VOCs for patients with SCD and transfusion requirements for patients with TDT.<\/p> \nAbout the CLIMB Studies<\/b><\/p> \nThe completed Phase 1\/2\/3 open-label studies, CLIMB-111 and CLIMB-121, were designed to assess the safety and efficacy of a single dose of CASGEVY in patients ages 12\u201335 years with TDT or with SCD and recurrent VOCs. Patients were followed for approximately two years after CASGEVY infusion in these studies. CLIMB-141 and CLIMB-151 are ongoing Phase 3 open-label studies, designed to assess the safety and efficacy of a single dose of exagamglogene autotemcel in patients ages 2\u201311 years with TDT or with SCD and recurrent VOCs. Enrollment and dosing are complete for the 5\u201311-year-old cohort in both studies.<\/p> \nEach patient in these studies is asked to participate in the ongoing long-term, open-label study, CLIMB-131. CLIMB-131 is designed to evaluate the long-term safety and efficacy of CASGEVY in patients with up to 15 years of follow-up after CASGEVY infusion.<\/p> \nU.S. INDICATIONS AND IMPORTANT SAFETY INFORMATION FOR CASGEVY<\/b><\/p> \nWHAT IS CASGEVY?<\/b><\/p> \nCASGEVY is a one-time therapy used to treat people ages 12 years and older with:<\/p> \n\u2022 sickle cell disease (SCD) who have frequent vaso-occlusive crises or VOCs<\/p> \n\u2022 beta thalassemia (\u03b2-thalassemia) who need regular blood transfusions<\/p> \nCASGEVY is made specifically for each patient, using the patient\u2019s own edited blood stem cells, and increases the production of a special type of hemoglobin called hemoglobin F (fetal hemoglobin or HbF). Having more HbF increases overall hemoglobin levels and has been shown to improve the production and function of red blood cells. This can eliminate VOCs in people with sickle cell disease and eliminate the need for regular blood transfusions in people with beta thalassemia.<\/p> \nIMPORTANT SAFETY INFORMATION<\/b><\/p> \nWhat is the most important information I should know about CASGEVY?<\/b><\/p> \nAfter treatment with CASGEVY, you will have fewer blood cells for a while until CASGEVY takes hold (engrafts) into your bone marrow. This includes low levels of platelets (cells that usually help the blood to clot) and white blood cells (cells that usually fight infections). Your doctor will monitor this and give you treatment as required. The doctor will tell you when blood cell levels return to safe levels.<\/p> \nYou may experience side effects associated with other medicines administered as part of the treatment regimen for CASGEVY. Talk to your physician regarding those possible side effects. Your healthcare provider may give you other medicines to treat your side effects.<\/p> \nHow will I receive CASGEVY?<\/b><\/p> \nYour healthcare provider will give you other medicines, including a conditioning medicine, as part of your treatment with CASGEVY. It\u2019s important to talk to your healthcare provider about the risks and benefits of all medicines involved in your treatment.<\/p> \nAfter receiving the conditioning medicine, it may not be possible for you to become pregnant or father a child. You should discuss options for fertility preservation with your healthcare provider before treatment.<\/p> \nSTEP 1:<\/b> Before CASGEVY treatment, a doctor will give you mobilization medicine(s). This medicine moves blood stem cells from your bone marrow into the blood stream. The blood stem cells are then collected in a machine that separates the different blood cells (this is called apheresis). This entire process may happen more than once. Each time, it can take up to one week.<\/p> \nDuring this step rescue cells are also collected and stored at the hospital. These are your existing blood stem cells and are kept untreated just in case there is a problem in the treatment process. If CASGEVY cannot be given after the conditioning <\/i>medicine, or if the modified blood stem cells do not take hold (engraft) in the body, these rescue cells will be given back to you. If you are given rescue cells, you will not have any treatment benefit from CASGEVY.<\/p> \nSTEP 2:<\/b> After they are collected, your blood stem cells will be sent to the manufacturing site where they are used to make CASGEVY. It may take up to 6 months from the time your cells are collected to manufacture and test CASGEVY before it is sent back to your healthcare provider.<\/p> \nSTEP 3:<\/b> Shortly before your stem cell transplant, your healthcare provider will give you a conditioning medicine for a few days in hospital. This will prepare you for treatment by clearing cells from the bone marrow, so they can be replaced with the modified cells in CASGEVY. After you are given this medicine, your blood cell levels will fall to very low levels. You will stay in the hospital for this step and remain in the hospital until after the infusion with CASGEVY.<\/p> \nSTEP 4:<\/b> One or more vials of CASGEVY will be given into a vein (intravenous infusion) over a short period of time.<\/p> \nAfter the CASGEVY infusion, you will stay in hospital so that your healthcare provider can closely monitor your recovery. This can take 4-6 weeks, but times can vary. Your healthcare provider will decide when you can go home.<\/p> \nWhat should I avoid after receiving CASGEVY?<\/b><\/p> \nWhat are the possible or reasonably likely side effects of CASGEVY?<\/b><\/p> \nThe most common side effects of CASGEVY include:<\/p> \nYour healthcare provider will test your blood to check for low levels of blood cells (including platelets and white blood cells). Tell your healthcare provider right away if you get any of the following symptoms:<\/p> \nThese are not all the possible side effects of CASGEVY. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.<\/p> \nGeneral information about the safe and effective use of CASGEVY<\/b><\/p> \nTalk to your healthcare provider about any health concerns.<\/p> \nPlease see full Prescribing Information<\/a> including Patient Information<\/a> for CASGEVY.<\/p> \nAbout Vertex<\/strong><\/p> \nVertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases and conditions. The company has approved therapies for cystic fibrosis, sickle cell disease, transfusion-dependent beta thalassemia and acute pain, and it continues to advance clinical and research programs in these areas. Vertex also has a robust clinical pipeline of investigational therapies across a range of modalities in other serious diseases where it has deep insight into causal human biology, including IgA nephropathy, neuropathic pain, APOL1-mediated kidney disease, primary membranous nephropathy, autosomal dominant polycystic kidney disease, type 1 diabetes, generalized myasthenia gravis, and myotonic dystrophy type 1.<\/p> \nVertex was founded in 1989 and has its global headquarters in Boston, with international headquarters in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia, Latin America and the Middle East. Vertex is consistently recognized as one of the industry's top places to work, including 16 consecutive years on Science magazine's Top Employers list and one of Fortune\u2019s 100 Best Companies to Work For. For company updates and to learn more about Vertex\u2019s history of innovation, visit www.vrtx.com<\/a> or follow us on LinkedIn<\/a>, Facebook<\/a>, Instagram<\/a>, YouTube<\/a> and X<\/a>.<\/p> \nVertex Special Note Regarding Forward-Looking Statements<\/strong><\/p> \nThis press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements made by Carmen Bozic, M.D., and Franco Locatelli, M.D., Ph.D., and statements regarding expectations for the transformative potential of CASGEVY in this age group, and expectations for the global regulatory submissions for younger children. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that data from the company's research and development programs may not support registration or further development of its potential medicines in a timely manner, or at all, due to safety, efficacy or other reasons, that CASGEVY may not receive regulatory approval for this age range on the expected timeline, or at all, and other risks listed under the heading \u201cRisk Factors\u201d in Vertex's most recent annual report and subsequent quarterly reports filed with the Securities and Exchange Commission at www.sec.gov<\/a> and available through the company's website at www.vrtx.com<\/a>. You should not place undue reliance on these statements, or the scientific data presented. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.<\/p> \n(VRTX-GEN)<\/strong><\/p> \nVertex Pharmaceuticals Incorporated<\/b><\/p> \nInvestors:\n\n
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