VRTX
Live News • 11h
Vertex Advances Pediatric Gene Editing and Launches New Therapies Beyond Cystic Fibrosis Vertex reported positive pivotal data for CASGEVY in children aged 5 to 11 years with severe sickle cell disease and transfusion-dependent beta thalassemia, with outcomes aligned to those seen in adults and adolescents.
The company is pursuing regulatory submissions to expand CASGEVY’s label to younger patients in the US, UK and Saudi Arabia, and shared early-age data for cystic fibrosis therapies ALYFTREK and TRIKAFTA.
Beyond cystic fibrosis, Vertex recently gained approval for suzetrigine, a first-in-class non-opioid acute pain drug, and is advancing late-stage programs in kidney disease, Type 1 diabetes and alpha-1 antitrypsin deficiency.
Taken together, Vertex is leaning on established cystic fibrosis cash flow while building out a broader portfolio in gene editing, pain and other serious diseases. This evolution may reshape its revenue mix over time if these programs clear regulatory and clinical hurdles.
Multiple late-stage trials, regulatory decisions and real-world use of newer products such as suzetrigine and CASGEVY introduce both execution risk and potential variability in future cash generation. Announcement • Jun 12
Vertex Pharmaceuticals Incorporated Presents New Data on Casgevy and Announces Additional Global Regulatory Submissions Vertex Pharmaceuticals Incorporated announced data demonstrating the clinical benefits of CASGEVY (exagamglogene autotemcel) in people ages 5 years and older living with severe sickle cell disease (SCD) or transfusion-dependent beta thalassemia (TDT). The results, from pivotal studies in children ages 5–11, show that the efficacy and safety outcomes in this age group are consistent with the transformative profile established in adult and adolescent patients. These data were presented at the European Hematology Association (EHA) Congress and simultaneously published in the New England Journal of Medicine (NEJM). Data from an interim analysis of the CLIMB-151 and CLIMB-141 studies highlight the transformative potential CASGEVY can provide to children ages 5–11 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. In the Phase 3 CLIMB-151 clinical study of children with severe SCD, all 11 patients dosed are free from VOCs and all 8 out of 8 (100%) patients with sufficient follow-up achieved the primary endpoint of being free from VOCs for at least 12 consecutive months (VF12). Of children achieving VF12, the mean (min, max) duration VOC-free was 19.0 (13.2, 30.1) months. In the Phase 3 CLIMB-141 clinical study of children with TDT, 15 patients have been dosed with CASGEVY, and all 8 out of 8 (100%) patients with sufficient follow-up achieved the primary endpoint of transfusion independence for at least 12 consecutive months while maintaining a weighted average hemoglobin of at least 9 g/dL (TI12). All children who achieved TI12 remained so throughout the follow-up; the mean (min, max) duration transfusion independence was 23.4 (13.3, 28.5) months. The safety profile of CASGEVY in younger patients is consistent with myeloablative conditioning and autologous transplant, as established in clinical studies in older patients with SCD and TDT. As previously disclosed, there was one death, not related to CASGEVY, in a child with TDT who developed severe veno-occlusive disease from busulfan conditioning. Consistent with studies in older patients, children with severe SCD and TDT treated with CASGEVY have durable and clinically relevant increases in fetal hemoglobin (HbF) and stable allelic editing. CASGEVY 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’s 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. The use of CASGEVY in children ages 5–11 years is investigational. CASGEVY is a non-viral, ex vivo CRISPR/Cas9 gene-edited cell therapy for eligible patients with SCD or TDT, in which a patient’s own hematopoietic stem and progenitor cells are edited at the erythroid specific enhancer region of the BCL11A 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. The 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 12–35 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 2–11 years with TDT or with SCD and recurrent VOCs. Enrollment and dosing are complete for the 5–11-year-old cohort in both studies. Each 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. CASGEVY is a one-time therapy used to treat people ages 12 years and older with sickle cell disease (SCD) who have frequent vaso-occlusive crises or VOCs and beta thalassemia (ß-thalassemia) who need regular blood transfusions. CASGEVY is made specifically for each patient, using the patient’s 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. After 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). The doctor will tell you when blood cell levels return to safe levels. You may experience side effects associated with other medicines administered as part of the treatment regimen for CASGEVY. Your healthcare provider may give you other medicines to treat your side effects. Your healthcare provider will give you other medicines, including a conditioning medicine, as part of your treatment with CASGEVY. After 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. 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). Announcement • Jun 06
Vertex Pharmaceuticals Incorporated Presents New Data On ALYFTREK And TRIKAFTA At European Cystic Fibrosis Conference Vertex Pharmaceuticals Incorporated announced data demonstrating the potentially transformative impact of treating cystic fibrosis (CF) with ALYFTREK (vanzacaftor/tezacaftor/deutivacaftor) in children ages 2 to 5, as well as data from 96-week interim analyses of two open-label extension studies of ALYFTREK in children 6 to 11 years and people 12 years and older demonstrating the long-term safety and efficacy profile of the medicine. The data, presented at the European Cystic Fibrosis Conference, show children ages 2 to 5 with vanzacaftor/tezacaftor/deutivacaftor-responsive genotypes including those who are homozygous for the F508del mutation (F/F) and those who have F508del/minimal function mutations (F/MF)on ALYFTREK had further improvement in CFTR function from a TRIKAFTA baseline as measured by sweat chloride (SwCl), with 65% having achieved SwCl <30 mmol/L after treatment with ALYFTREK. Vertex also presented Phase 3 data of children ages 1 to <2 with TRIKAFTA (elexacaftor/tezacaftor/ivacaftor). Vertex plans to submit for global regulatory approvals of ALYFTREK in children ages 2 to 5 in the first half of 2026, and the company has begun global regulatory submissions for TRIKAFTA in children ages 1 to <2. “A Phase 3 open-label clinical trial of vanzacaftor/tezacaftor/deutivacaftor in children aged 2-5 years with cystic fibrosis”was presented as a late-breaking abstract and oral presentation in the “Late-Breaking Science” session on June 5 from 5:00 p.m. to 6:30 p.m. GMT+1. Data from 67 children who all completed the 24-week, Phase 3, open-label study show that ALYFTREK was generally safe and well tolerated, consistent with the established safety profile. The primary endpoint of the study was safety and tolerability. Treatment with ALYFTREK resulted in a rapid, clinically meaningful improvement in CFTR function with a mean reduction in sweat chloride from a baseline on TRIKAFTA of -9.6 mmol/L (95% CI -12.1 to -7.0) through Week 24, with 92% of children achieving SwCl concentrations of <60 mmol/L (the diagnostic threshold for CF), and 65% of children reaching SwCl values of <30 mmol/L. These improvements in CFTR function surpass those seen in trials with any other CFTR modulator in this age group. Vertex also presented multiple abstracts on clinical and real-world evidence on ALYFTREK and TRIKAFTA as listed below. These abstracts will be published in the Journal of Cystic Fibrosis: “Long-term safety and efficacy of vanzacaftor/tezacaftor/deutivacaftor in people with cystic fibrosis aged 12 years and older: 96-week interim analysis from an open-label extension study.” (Poster 143) “Long-term safety and efficacy of vanzacaftor/tezacaftor/deutivacaftor in children with cystic fibrosis aged 6 years and older: 96-week interim analysis from an open-label extension study”; also presented as an oral presentation (WS01.3) during the symposium “Clinical and functional impact of highly effective modulators” on June 4 from 3:00–4:30 p.m. GMT+1. “Demographic and clinical characteristics of children with CF aged 2-5 years initiating ELX/TEZ/IVA in LONGITUDE — a UK CF Registry observational study.” (Poster P432). “A Phase 3, 24-Week, Open-Label Study of Elexacaftor/Tezacaftor/Ivacaftor in Children with Cystic Fibrosis 12 to <24 Months of Age” (WS01.2)was featured in an oral presentation as part of the symposium “Clinical and functional impact of highly effective modulators” on June 4 from 3:00–4:30 p.m. GMT+1 and the abstract will be published in the Journal of Cystic Fibrosis:. Results from a 24-week, Phase 3, open-label study of TRIKAFTA in 54 enrolled children aged 12 to <24 months was presented. The primary endpoint was safety and tolerability. TRIKAFTA was generally safe and well tolerated; the safety data are consistent with the established safety profile. Treatment with TRIKAFTA in this age group resulted in rapid, statistically significant and clinically meaningful decrease in SwCl, with a mean reduction of -71.8 mmol/L from a baseline without CFTR modulator treatment through Week 24, with 98.0% of children achieving concentrations <60 mmol/L and 68.6% reaching <30 mmol/L. The uses of ALYFTREK in children with CF 2 to 5 years old, and TRIKAFTA in children with CF 1 to <2 years old, are investigational. 
