Lumacaftor

Presentations showcase diversity and momentum of Pfizer’s growing portfolio of vaccines and anti-infectives NEW YORK--(BUSINESS WIRE)-- Pfizer Inc. (NYSE: PFE) will present data across its infectious disease portfolio at the upcoming IDWeek 2023 held in Boston from October 11-15, 2023. Data from 45 abstracts will highlight the advances Pfizer is making in helping prevent and treat certain infectious diseases, particularly respiratory illnesses. This will include research featured in the IDWeek press program assessing the potential public health impact of ABRYSVO™ (Respiratory Syncytial Virus Vaccine), the company’s bivalent respiratory syncytial virus prefusion F (RSVpreF) vaccine for maternal immunization to protect infants against RSV illness. “The data at this year’s IDWeek highlight Pfizer’s commitment to transforming respiratory health, an area where we are proud to have delivered several innovative vaccine and treatment options over the last three years. We also continue to actively investigate infectious diseases more broadly, with a diverse pipeline of innovative vaccine and anti-infective treatment candidates,” said Annaliesa Anderson, Ph.D., Senior Vice President and Head, Vaccine Research and Development, Pfizer. “With our unique scientific capabilities and vaccine technology platforms, we are unwavering in the fight against the world’s most pressing disease challenges. We are excited to present our latest clinical results that support our growing infectious disease portfolio for the purpose of improving global health outcomes.” "The burden of respiratory illnesses on global public health is significant. It is particularly relevant as we enter the winter respiratory season in the Northern Hemisphere and once again face the threat of a possible tripledemic with influenza, RSV and COVID-19 co-circulating,” said Luis Jodar, Ph. D., Chief Medical Affairs Officer, Vaccines/Antivirals and Evidence Generation, Pfizer. “At IDWeek, we are pleased to share real-world data that will help educate healthcare professionals about the role Pfizer’s vaccines and treatments can play in helping mitigate the impact of certain respiratory infections during this critical period.” Pfizer's participation at IDWeek 2023 comprises breakthrough research from across the company’s robust infectious disease portfolio, covering RSV, COVID-19, pneumococcal disease, Lyme disease, meningococcal disease, multidrug-resistant gram-negative bacterial infections and more. Eight abstracts have been selected for oral presentation, including data on the successful coadministration of ABRYSVO with an influenza vaccine in older adults and results from the Phase 3 REVISIT study of Pfizer’s investigational antibiotic combination aztreonam-avibactam (ATM-AVI) candidate. Data highlighting the real-world effectiveness of Pfizer’s FDA-approved COVID-19 oral treatment for adults, PAXLOVID™ (nirmatrelvir tablets; ritonavir tablets), will also be featured at the meeting. A complete list of Pfizer-sponsored accepted abstracts is available here. Details for Pfizer-sponsored oral presentations are below: Title/ Abstract Number Presenting Name/Type Date/ Time EDT Location IDWEEK PRESS PROGRAM 1942 - Potential Public Health Impact of Bivalent Respiratory Syncytial Virus Prefusion F (RSVpreF) Maternal Vaccine for Prevention of RSV Among US Infants Amy W. Law, PharmD Oct 13 2:15 - 2:30 PM US ET 104 ABC ORAL PRESENTATIONS 1019 - Respiratory Syncytial Virus (RSV) Diagnoses in Hospitalized Patients Increases when Additional Specimen Types are Added to Nasopharyngeal Swab: Results from the RSV in Adults Multispecimen Study Elizabeth Begier, MD, MPH Oct 12 3:15 - 3:33 PM US ET 104 ABC 1941 - Coadministration of Bivalent Respiratory Syncytial Virus (RSVpreF) Vaccine with Influenza Vaccine in Older Adults James Baber, MBChB, MPH Oct 13 2:00 - 2:15 PM US ET 104 ABC 1944 - Tolerability and Safety of 20-Valent Pneumococcal Conjugate Vaccine (PCV20) in Infants and Older Children in Global Studies Kathleen McElwee, MD, MPH Oct 13 2:45 - 3:00 PM US ET 104 ABC 1948 - Rise of Candida auris Infections Worldwide and Trends on the Activity of Fosmanogepix and Comparator Agents Against C. auris Causing Invasive Infections Cecilia G. Carvalhaes, MD, PhD Oct 13 2:30 - 2:45 PM US ET 102 AB 1949 – Activity of Aztreonam-Avibactam Against Enterobacterales Resistant to Recently Approved Beta-Lactamase Inhibitor Combinations Collected Worldwide (ex-US: 2020-2022) Helio S. Sader, MD, PhD, FIDSA Oct 13 2:45 - 3:00 PM US ET 102 AB 2890 - Pharmacokinetics, Safety, and Efficacy of Ceftazidime-Avibactam in Neonates and Young Infants with Bacterial Infections: Results from a Phase 2a, 2-part, Open-label, Non-randomized, Multicenter Trial Richard D England, MD Oct 14 2:05 - 2:15 PM US ET 254 AB 2893 - Efficacy and Safety of Aztreonam-Avibactam for the Treatment of Serious Infections Due to Gram-Negative Bacteria, Including Metallo-β-Lactamase-Producing Pathogens: Phase 3 REVISIT Study Yehuda Carmeli, MD MPH Oct 14 2:35 - 2:45 PM US ET 254 AB INDICATIONS AND IMPORTANT SAFETY INFORMATION FOR ABRYSVO U.S. INDICATIONS ABRYSVOTM is a vaccine indicated for: the prevention of lower respiratory tract disease (LRTD) caused by respiratory syncytial virus (RSV) in people 60 years of age and older pregnant individuals at 32 through 36 weeks gestational age for the prevention of LRTD and severe LRTD caused by RSV in infants from birth through 6 months of age IMPORTANT SAFETY INFORMATION ABRYSVO should not be given to anyone with a history of severe allergic reaction (eg, anaphylaxis) to any of its components For pregnant individuals: to avoid the potential risks of preterm birth, ABRYSVO should be given during 32 through 36 weeks gestational age Fainting can happen after getting injectable vaccines, including ABRYSVO. Precautions should be taken to avoid falling and injury during fainting Adults with weakened immune systems, including those receiving medicines that suppress the immune system, may have a reduced immune response to ABRYSVO Vaccination with ABRYSVO may not protect all people In adults 60 years of age and older, the most common side effects (≥10%) were fatigue, headache, pain at the injection site, and muscle pain In pregnant individuals, the most common side effects (≥10%) were pain at the injection site, headache, muscle pain, nausea In clinical trials where ABRYSVO was compared to placebo, infants born to pregnant individuals experienced low birth weight (5.1% ABRYSVO versus 4.4% placebo) and jaundice (7.2% ABRYSVO versus 6.7% placebo View the full Prescribing Information. INDICATION, AUTHORIZED USE AND IMPORTANT SAFETY INFORMATION FOR PAXLOVID U.S. Indication PAXLOVID is indicated for the treatment of mild-to-moderate coronavirus disease 2019 (COVID-19) in adults who are at high risk for progression to severe COVID-19, including hospitalization or death. Limitations of Use PAXLOVID is not approved for use as pre-exposure or post-exposure prophylaxis for prevention of COVID-19. U.S. FDA Emergency Use Authorization Statement The U.S. Food and Drug Administration (FDA) has issued an Emergency Use Authorization (EUA) for the emergency use of PAXLOVID for the treatment of adults and pediatric patients (12 years of age and older weighing at least 40 kg) with mild to moderate coronavirus disease 2019 (COVID-19) and who are at high risk for progression to severe COVID-19, including hospitalization or death. PAXLOVID has not been approved, but has been authorized for emergency use by FDA under an EUA, for the treatment of mild-to-moderate COVID-19 in pediatric patients (12 years of age and older weighing at least 40 kg) who are at high risk for progression to severe COVID-19, including hospitalization or death. The emergency use of PAXLOVID is only authorized for the duration of the declaration that circumstances exist justifying the authorization of the emergency use of drugs and biological products during the COVID-19 pandemic under Section 564(b)(1) of the Act, 21 U.S.C. § 360bbb-3(b)(1), unless the declaration is terminated or authorization revoked sooner. IMPORTANT SAFETY INFORMATION WARNING: SIGNIFICANT DRUG INTERACTIONS WITH PAXLOVID PAXLOVID includes ritonavir, a strong CYP3A inhibitor, which may lead to greater exposure of certain concomitant medications, resulting in potentially severe, life-threatening, or fatal events Prior to prescribing PAXLOVID: 1) Review all medications taken by the patient to assess potential drug-drug interactions with a strong CYP3A inhibitor like PAXLOVID and 2) Determine if concomitant medications require a dose adjustment, interruption, and/or additional monitoring Consider the benefit of PAXLOVID treatment in reducing hospitalization and death, and whether the risk of potential drug-drug interactions for an individual patient can be appropriately managed PAXLOVID is contraindicated in patients with a history of clinically significant hypersensitivity reactions (eg, toxic epidermal necrolysis or Stevens-Johnson syndrome) to its active ingredients (nirmatrelvir or ritonavir) or any other components of the product. If signs and symptoms of a clinically significant hypersensitivity reaction or anaphylaxis occur, immediately discontinue PAXLOVID and initiate appropriate medications and/or supportive care. PAXLOVID is contraindicated with drugs that are primarily metabolized by CYP3A and for which elevated concentrations are associated with serious and/or life-threatening reactions and drugs that are strong CYP3A inducers where significantly reduced nirmatrelvir or ritonavir plasma concentrations may be associated with the potential for loss of virologic response and possible resistance. There are certain other drugs for which concomitant use with PAXLOVID should be avoided and/or dose adjustment, interruption, or therapeutic monitoring is recommended. Drugs listed here are a guide and not considered a comprehensive list of all drugs that may be contraindicated with PAXLOVID. The healthcare provider should consult other appropriate resources such as the prescribing information for the interacting drug for comprehensive information on dosing or monitoring with concomitant use of a strong CYP3A inhibitor like PAXLOVID. Drugs that are primarily metabolized by CYP3A for which elevated concentrations are associated with serious and/or life-threatening reactions: Alpha 1-adrenoreceptor antagonist: alfuzosin Antianginal: ranolazine Antiarrhythmic: amiodarone, dronedarone, flecainide, propafenone, quinidine Anti-gout: colchicine (in patients with renal and/or hepatic impairment) Antipsychotics: lurasidone, pimozide Benign prostatic hyperplasia agents: silodosin Cardiovascular agents: eplerenone, ivabradine Ergot derivatives: dihydroergotamine, ergotamine, methylergonovine HMG-CoA reductase inhibitors: lovastatin, simvastatin (these drugs can be temporarily discontinued to allow PAXLOVID use) Immunosuppressants: voclosporin Microsomal triglyceride transfer protein inhibitor: lomitapide Migraine medications: eletriptan, ubrogepant Mineralocorticoid receptor antagonists: finerenone Opioid antagonists: naloxegol PDE5 inhibitor: sildenafil (Revatio®) when used for pulmonary arterial hypertension Sedative/hypnotics: triazolam, oral midazolam Serotonin receptor 1A agonist/serotonin receptor 2A antagonist: flibanserin Vasopressin receptor antagonists: tolvaptan Drugs that are strong CYP3A inducers: PAXLOVID cannot be started immediately after discontinuation of any of the following medications due to the delayed offset of the recently discontinued CYP3A inducer: Anticancer drugs: apalutamide Anticonvulsant: carbamazepine, phenobarbital, primidone, phenytoin Antimycobacterials: rifampin, rifapentine Cystic fibrosis transmembrane conductance regulator potentiators: lumacaftor/ivacaftor Herbal Products: St. John’s Wort (hypericum perforatum) Risk of Serious Adverse Reactions Due to Drug Interactions: Initiation of PAXLOVID, which contains ritonavir, a strong CYP3A inhibitor, in patients receiving medications metabolized by CYP3A or initiation of medications metabolized by CYP3A in patients already receiving PAXLOVID, may increase plasma concentrations of medications metabolized by CYP3A. Medications that induce CYP3A may decrease concentrations of PAXLOVID. These interactions may lead to: Clinically significant adverse reactions, potentially leading to severe, life-threatening, or fatal events from greater exposures of concomitant medications Loss of therapeutic effect of PAXLOVID and possible development of viral resistance Severe, life-threatening, and/or fatal adverse reactions due to drug interactions have been reported in patients treated with PAXLOVID. The most commonly reported concomitant medications resulting in serious adverse reactions were calcineurin inhibitors (eg, tacrolimus, cyclosporine), followed by calcium channel blockers. Hepatotoxicity: Hepatic transaminase elevations, clinical hepatitis, and jaundice have occurred in patients receiving ritonavir. Therefore, caution should be exercised when administering PAXLOVID to patients with pre-existing liver diseases, liver enzyme abnormalities, or hepatitis. Because nirmatrelvir is coadministered with ritonavir, there may be a risk of HIV-1 developing resistance to HIV protease inhibitors in individuals with uncontrolled or undiagnosed HIV-1 infection. The most common adverse reactions in the PAXLOVID group (≥1%) that occurred at a greater frequency than in the placebo group were dysgeusia (5% and <1%, respectively) and diarrhea (3% and 2%, respectively). The following adverse reactions have been identified during use of PAXLOVID under Emergency Use Authorization: Immune System Disorders: Anaphylaxis, hypersensitivity reactions Skin and Subcutaneous Tissue Disorders: Toxic epidermal necrolysis, Stevens-Johnson syndrome Nervous System Disorders: Headache Vascular Disorders: Hypertension Gastrointestinal Disorders: Abdominal pain, nausea, vomiting General Disorders and Administration Site Conditions: Malaise PAXLOVID is a strong inhibitor of CYP3A, and an inhibitor of CYP2D6, P-gp, and OATP1B1. Coadministration of PAXLOVID with drugs that are primarily metabolized by CYP3A and CYP2D6 or are transported by P-gp or OATP1B1 may result in increased plasma concentrations of such drugs and increase the risk of adverse events. Coadministration with other CYP3A substrates may require a dose adjustment or additional monitoring. Pregnancy: Available data on the use of nirmatrelvir during pregnancy are insufficient to evaluate for a drug-associated risk of major birth defects, miscarriage, or adverse maternal or fetal outcomes. Published observational studies on ritonavir use in pregnant women have not identified an increase in the risk of major birth defects. Published studies with ritonavir are insufficient to identify a drug-associated risk of miscarriage. There are maternal and fetal risks associated with untreated COVID-19 in pregnancy. Lactation: There are no available data on the presence of nirmatrelvir in human or animal milk, the effects on the breastfed infant, or the effects on milk production. A transient decrease in body weight was observed in the nursing offspring of rats administered nirmatrelvir. Limited published data report that ritonavir is present in human milk. There is no information on the effects of ritonavir on the breastfed infant or the effects of the drug on milk production. The developmental and health benefits of breastfeeding should be considered along with the mother's clinical need for PAXLOVID and any potential adverse effects on the breastfed infant from PAXLOVID or from the underlying maternal condition. Contraception: Use of ritonavir may reduce the efficacy of combined hormonal contraceptives. Advise patients using combined hormonal contraceptives to use an effective alternative contraceptive method or an additional barrier method of contraception. Pediatrics: The optimal dose of PAXLOVID has not been established in pediatric patients. Systemic exposure of nirmatrelvir increases in renally impaired patients with increase in the severity of renal impairment. No dosage adjustment is recommended in patients with mild renal impairment. Reduce the dose of PAXLOVID in patients with moderate renal impairment (eGFR ≥30 to <60 mL/min). PAXLOVID is not recommended in patients with severe renal impairment (eGFR <30 mL/min) or in patients with end-stage renal disease (eGFR <15 mL/min). PAXLOVID is not recommended for use in patients with severe hepatic impairment (Child-Pugh Class C). Please see Full Prescribing Information, including BOXED WARNING and Patient Information Click for Fact Sheets: For Consumers: EUA Fact sheet for Patients, Parents, and Caregivers For Healthcare Professionals: EUA Fact Sheet for HCPs INDICATIONS AND IMPORTANT SAFETY INFORMATION FOR PREVNAR 20 U.S. INDICATIONS PREVNAR 20® is a vaccine approved for: the prevention of invasive disease caused by 20 Streptococcus pneumoniae strains (1, 3, 4, 5, 6A, 6B, 7F, 8, 9V, 10A, 11A, 12F, 14, 15B, 18C, 19A, 19F, 22F, 23F, and 33F) in individuals 6 weeks and older. the prevention of otitis media (middle ear infection) caused by 7 of the 20 strains in individuals 6 weeks through 5 years. IMPORTANT SAFETY INFORMATION PREVNAR 20 should not be given to anyone who has had a severe allergic reaction to any component of PREVNAR 20 or to diphtheria–toxoid-containing vaccine. Individuals with weakened immune systems may have a lower immune response. Safety data are not available for these groups. A temporary pause in breathing after getting the vaccine has been observed in some infants who were born prematurely. For premature infants, talk to your doctor about the infant's medical status when deciding to get vaccinated with PREVNAR 20. In individuals 2, 4, 6, and 12 through 15 months of age vaccinated with a 4-dose schedule, the most common side effects reported at a rate of >10% were irritability, pain at the injection site, drowsiness, decreased appetite and injection site redness, injection site swelling, and fever. In individuals 15 months through 17 years of age vaccinated with a single dose, the most common side effects reported at a rate of >10% were irritability, pain at the injection site, drowsiness, fatigue and muscle pain, decreased appetite, injection site swelling and injection site redness, headache, and fever. Ask your doctor about the risks and benefits of PREVNAR 20. Only a doctor can decide if PREVNAR 20 is right for your child. View the full Prescribing Information. About Pfizer: Breakthroughs That Change Patients’ Lives At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products, including innovative medicines and vaccines. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time. Consistent with our responsibility as one of the world's premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world. For more than 170 years, we have worked to make a difference for all who rely on us. We routinely post information that may be important to investors on our website at . In addition, to learn more, please visit us on and follow us on Twitter at @Pfizer and @Pfizer News, LinkedIn, YouTube and like us on Facebook at Facebook.com/Pfizer. Disclosure Notice The information contained in this release is as of October 5, 2023. Pfizer assumes no obligation to update forward-looking statements contained in this release as the result of new information or future events or developments. This release contains forward-looking information about Pfizer’s infectious disease pipeline, in-line products and product candidates, including ABRYSVO (RSVpreF), Pfizer’s aztreonam-avibactam (ATM-AVI) candidate and PAXLOVID, including their potential benefits, that involves substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. Risks and uncertainties include, among other things, uncertainties regarding the commercial success of ABRYSVO (RSVpreF), Pfizer’s ATM-AVI candidate, PAXLOVID or any of Pfizer’s other infectious disease products or product candidates; the uncertainties inherent in research and development, including the ability to meet anticipated clinical endpoints, commencement and/or completion dates for our clinical trials, regulatory submission dates, regulatory approval dates and/or launch dates, as well as risks associated with pre-clinical and clinical data (including Phase 1/2/3 or Phase 4 or pre-clinical data for ABRYSVO (RSVpreF), Pfizer’s ATM-AVI candidate, PAXLOVID or any of Pfizer’s other infectious disease products or product candidates, including the data discussed in this release) in any of our studies in pediatrics, adolescents, or adults or real world evidence, including the possibility of unfavorable new pre-clinical, clinical or safety data and further analyses of existing pre-clinical, clinical or safety data; risks associated with interim data, including the risk that final results from the Phase 3 trials for RSVpreF could differ from the interim data; the risk that clinical trial data are subject to differing interpretations and assessments by regulatory authorities; the ability to produce comparable clinical or other results for ABRYSVO (RSVpreF), Pfizer’s ATM-AVI candidate, PAXLOVID or any of Pfizer’s other infectious disease products or product candidates, including efficacy, safety and tolerability pro to date, in additional analyses of the Phase 3 trial and additional studies, in real world data studies or in larger, more diverse populations following commercialization; the ability of ABRYSVO (RSVpreF) and PAXLOVID to be effective against emerging virus variants; the risk that the use of ABRYSVO (RSVpreF), Pfizer’s ATM-AVI candidate, PAXLOVID or any of Pfizer’s other infectious disease products or product candidates will lead to new information about efficacy, safety, or other developments, including the risk of additional adverse reactions, some of which may be serious; the risk that pre-clinical and clinical trial data are subject to differing interpretations and assessments, including during the peer review/publication process, in the scientific community generally, and by regulatory authorities; whether and when additional data from ABRYSVO (RSVpreF), Pfizer’s ATM-AVI candidate, PAXLOVID or any of Pfizer’s other infectious disease products or product candidates will be published in scientific journal publications and, if so, when and with what modifications and interpretations; whether regulatory authorities will be satisfied with the design of and results from our clinical studies; whether and when drug applications or submissions to request emergency use or conditional marketing authorizations for any potential indications for ABRYSVO (RSVpreF), Pfizer’s ATM-AVI candidate, PAXLOVID or any of Pfizer’s other infectious disease products or product candidates may be filed in any jurisdictions and if obtained, whether or when such emergency use authorization or licenses will expire or terminate; whether and when any applications that may be pending or filed for ABRYSVO (RSVpreF), Pfizer’s ATM-AVI candidate, PAXLOVID or any of Pfizer’s other infectious disease products or product candidates (including any requested amendments to the emergency use or conditional marketing authorizations) may be approved by regulatory authorities, which will depend on myriad factors, including making a determination as to whether the product's benefits outweigh its known risks and determination of the product's efficacy and, if approved, whether ABRYSVO (RSVpreF), Pfizer’s ATM-AVI candidate, PAXLOVID or any of Pfizer’s other infectious disease products or product candidates will be commercially successful; intellectual property and other litigation; decisions by regulatory authorities impacting labeling, manufacturing processes, safety and/or other matters that could affect the availability or commercial potential of ABRYSVO (RSVpreF), Pfizer’s ATM-AVI candidate, PAXLOVID or any of Pfizer’s other infectious disease products or product candidates, including the authorization or approval of products or therapies developed by other companies; the risk that demand for any of our products may be reduced, no longer exist or not meet expectations, which may lead to excess inventory on-hand and/or in the channel, inventory write-offs or reduced revenues; challenges related to and uncertainties regarding the timing of a transition to the commercial market for any of our products; uncertainties related to the public’s adherence to vaccines and boosters; risks related to our ability to achieve our revenue forecasts for any of Pfizer’s infectious disease products or product candidates; the risk that other companies may produce superior or competitive products; risks related to the availability of raw materials to manufacture or test Pfizer’s infectious disease products or product candidates; challenges related to our vaccine’s formulation, dosing schedule and attendant storage, distribution and administration requirements, including risks related to storage and handling after delivery by Pfizer; the risk that we may not be able to successfully develop other vaccine formulations, booster doses or potential future annual boosters or re-vaccinations or new variant-based or next generation vaccines or potential combination respiratory vaccines; whether and when additional supply or purchase agreements will be reached or existing agreements will be completed or renegotiated; uncertainties regarding the ability to obtain recommendations from vaccine advisory or technical committees and other public health authorities regarding ABRYSVO (RSVpreF) or any of Pfizer’s other infectious disease products or product candidates and uncertainties regarding the commercial impact of any such recommendations; pricing and access challenges; challenges related to public confidence in, or awareness of ABRYSVO (RSVpreF), Pfizer’s ATM-AVI candidate, PAXLOVID or any of Pfizer’s other infectious disease products or product candidates; uncertainties regarding the impact of COVID-19 on our business, operations and financial results; and competitive developments. A further description of risks and uncertainties can be found in Pfizer’s Annual Report on Form 10-K for the fiscal year ended December 31, 2022, and in its subsequent reports on Form 10-Q, including in the sections thereof captioned “Risk Factors” and “Forward-Looking Information and Factors That May Affect Future Results”, as well as in its subsequent reports on Form 8-K, all of which are filed with the U.S. Securities and Exchange Commission and available at and .

-Interim results of largest real-world study of TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) showed sustained improvement in lung function, reduction in pulmonary exacerbations frequency and lower rates of lung transplant and death for people with cystic fibrosis- - Twelve presentations add to the body of evidence supporting the use of CFTR modulators for all eligible people with cystic fibrosis- BOSTON--(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that 12 scientific abstracts on the company’s portfolio of cystic fibrosis (CF) medicines were presented at this year’s European Cystic Fibrosis Society's (ECFS) 46th European Cystic Fibrosis Conference held June 7-10, 2023, in Vienna, Austria. Together, the data presented show the long-term benefits of treatment with CFTR modulators as well as the importance of treating the underlying cause of CF as early in life as possible. Key data presented at this year’s conference are highlighted below. Vertex presented an interim analysis (IA) of a registry-based study of real-world data collected from people with CF and treated with TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor), also known in the European Union and in the U.K. as KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor, including over 16,000 people with CF from the Cystic Fibrosis Foundation Patient Registry (CFFPR) and nearly 3,000 from the German CF Registry. This ongoing five-year post-authorization study (abstract WS16.03) is the largest real-world study of people with CF treated with TRIKAFTA® to date. The IA showed clinically significant disease-modifying benefits for TRIKAFTA®, including improved lung function and a 79% reduction of pulmonary exacerbations in the U.S. and 83% in Germany overall compared to pre-TRIKAFTA® baseline. The rate of death was 72% lower in the U.S. and 82% lower in Germany, the rate of lung transplant was 85% lower in the U.S. and 100% lower in Germany, compared to 2019 (pre-TRIKAFTA®) U.S. CFFPR and German CF Registry populations. No new safety concerns were identified. Vertex also presented final results of the nearly four-year TRIKAFTA® open-label follow-up study of the Phase 3 pivotal trials in people with CF ages 12 years and older with at least one F508del mutation in the CFTR gene (Late Breaking Science; Workshop 15). The results of this study are unprecedented, showing for the first time that treatment with TRIKAFTA® resulted in no decline in lung function over a four-year period. “CFTR modulators markedly improve clinical outcomes of people living with CF as demonstrated in the large and growing quantity of data presented at ECFS this year,” said Professor Isabelle Fajac, Professor of Physiology, Cochin Hospital, Assistance Publique-Hôpitaux de Paris, Université Paris Cité, Paris, France. “The data on TRIKAFTA in particular demonstrate that this medicine improves lung function sustainably and in a real-world setting. It also reduces the risks of pulmonary exacerbation, death and lung transplant, and it is generally well tolerated.” Additional Presentations Other Vertex presentations at the conference this year include: Abstract EPS6.05 entitled “A Phase 3b study of the effects of elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) on cough and physical activity in people with cystic fibrosis (CF)” Abstract WS05.04 entitled “Safety and efficacy of ivacaftor (IVA) in children aged 1 to <4 months with cystic fibrosis assessed with an innovative clinical trial design” Late Breaking Science (Workshop 15) abstract entitled “LONGITUDE: An observational study of the long-term effectiveness of ivacaftor/tezacaftor/elexacaftor in people with cystic fibrosis using data from the United Kingdom Cystic Fibrosis Registry” Abstract WS16.02 entitled “Real-world (RW) clinical effectiveness of elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) in children with cystic fibrosis aged 6-11 years: interim results from the HELIO study” Abstract P117 entitled “A longitudinal study on the impact of ELX/TEZ/IVA treatment on quality of life in people with cystic fibrosis in the real world” Abstract P118 entitled “Real-world impact of ELX/TEZ/IVA on quality of life of children with CF aged 6-11 years and primary caregivers in the UK: MAGNIFY, a prospective, observational, noninterventional study” Abstract WS05.03 entitled “A Phase 3b study of the effects of elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) on glucose tolerance in people with cystic fibrosis (CF) and abnormal glucose metabolism” Abstract WS16.04 entitled “Benefits of lumacaftor/ivacaftor (LUM/IVA) initiation in children with CF aged 2 through 5 years: Interim results from an ongoing registry-based study” Abstract WS16.05 entitled “Long-Term Impact of Ivacaftor (IVA) in People with Cystic Fibrosis in Ireland” Late Breaking Science (Workshop 15) abstract entitled “Effects of elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) treatment on markers of inflammation in people with cystic fibrosis (CF)” About Cystic Fibrosis Cystic fibrosis (CF) is a rare, life-shortening genetic disease affecting more than 88,000 people globally. CF is a progressive, multi-organ disease that affects the lungs, liver, pancreas, GI tract, sinuses, sweat glands and reproductive tract. CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF, and these mutations can be identified by a genetic test. While there are many different types of CFTR mutations that can cause the disease, the vast majority of people with CF have at least one F508del mutation. CFTR mutations lead to CF by causing CFTR protein to be defective or by leading to a shortage or absence of CFTR protein at the cell surface. The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus, chronic lung infections and progressive lung damage that eventually leads to death for many patients. The median age of death is in the early 30s. About TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) In people with certain types of mutations in the CFTR gene, the CFTR protein is not processed or folded normally within the cell, and this can prevent the CFTR protein from reaching the cell surface and functioning properly. TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) is an oral medicine designed to increase the quantity and function of the CFTR protein at the cell surface. Elexacaftor and tezacaftor work together to increase the amount of mature protein at the cell surface. Ivacaftor, which is known as a CFTR potentiator, is designed to facilitate the ability of CFTR proteins to transport salt and water across the cell membrane. The combined actions of elexacaftor, tezacaftor and ivacaftor help hydrate and clear mucus from the airways. U.S. INDICATION AND IMPORTANT SAFETY INFORMATION FOR TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) TRIKAFTA is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients aged 2 years and older who have at least one copy of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or another mutation that is responsive to treatment with TRIKAFTA. Patients should talk to their doctor to learn if they have an indicated CF gene mutation. It is not known if TRIKAFTA is safe and effective in children under 2 years of age. Before taking TRIKAFTA, patients should tell their doctor about all of their medical conditions, including if they: have kidney problems, have or have had liver problems, are pregnant or plan to become pregnant because it is not known if TRIKAFTA will harm an unborn baby, or are breastfeeding or planning to breastfeed because it is not known if TRIKAFTA passes into breast milk. Tell your doctor about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. TRIKAFTA may affect the way other medicines work, and other medicines may affect how TRIKAFTA works. The dose of TRIKAFTA may need to be adjusted when taken with certain medicines. Patients should ask their doctor or pharmacist for a list of these medicines if they are not sure. Patients should especially tell their doctor if they take: antibiotics such as rifampin or rifabutin; seizure medicines such as phenobarbital, carbamazepine, or phenytoin; St. John’s wort; antifungal medicines including ketoconazole, itraconazole, posaconazole, voriconazole, or fluconazole; antibiotics including telithromycin, clarithromycin, or erythromycin. Patients should avoid food or drink that contains grapefruit while they are taking TRIKAFTA. TRIKAFTA can cause serious side effects, including: Liver damage and worsening of liver function in people with severe liver disease that can be serious and may require transplantation. Liver damage has also happened in people without liver disease. High liver enzymes in the blood, which is a common side effect in people treated with TRIKAFTA. These can be serious and may be a sign of liver injury. The patient’s doctor will do blood tests to check their liver before they start TRIKAFTA, every 3 months during the first year of taking TRIKAFTA, and every year while taking TRIKAFTA. Patients should call their doctor right away if they have any of the following symptoms of liver problems: pain or discomfort in the upper right stomach (abdominal) area; yellowing of the skin or the white part of the eyes; loss of appetite; nausea or vomiting; dark, amber-colored urine. Abnormality of the eye lens (cataract) has been noted in some children and adolescents treated with TRIKAFTA. If the patient is a child or adolescent, their doctor should perform eye examinations before and during treatment with TRIKAFTA to look for cataracts. The most common side effects of TRIKAFTA include headache, upper respiratory tract infection (common cold) including stuffy and runny nose, stomach (abdominal) pain, diarrhea, rash, increase in liver enzymes, increase in a certain blood enzyme called creatine phosphokinase, flu (influenza), inflamed sinuses, and increase in blood bilirubin. Patients should tell their doctor if they have any side effect that bothers them or that does not go away. These are not all the possible side effects of TRIKAFTA. For more information, patients should ask their doctor or pharmacist. Please click here to see the full U.S. Prescribing Information for TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor). About Vertex Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) — a rare, life-threatening genetic disease — and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust clinical pipeline of investigational small molecule, mRNA, cell and genetic therapies (including gene editing) in other serious diseases where it has deep insight into causal human biology, including sickle cell disease, beta thalassemia, APOL1-mediated kidney disease, acute and neuropathic pain, type 1 diabetes and alpha-1 antitrypsin deficiency. Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 13 consecutive years on Science magazine's Top Employers list and one of Fortune’s 100 Best Companies to Work For. For company updates and to learn more about Vertex's history of innovation, visit or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram. Special Note Regarding Forward-Looking Statements This 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 Professor Isabelle Fajac in this press release, and statements regarding the potential benefits, safety and efficacy of our medicines, and our plans to present data about our medicines at the ECFS European Cystic Fibrosis Conference. 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 development programs may not support registration, approval or further development of its compounds due to safety, efficacy or other reasons, risks related to approval and commercialization of our medicines, and other risks listed under the heading “Risk Factors” in Vertex's most recent annual report and subsequent quarterly reports filed with the Securities and Exchange Commission (SEC) and available through the company's website at and on the SEC’s website at . 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. (VRTX-GEN)